Advocacy

07 Aug 2013
Sticky Post By Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

Local man fights for Alberta child’s health

Sticky Post By On 7 August, 2013

By Jeff Gard, Northumberland Today

Andrew McFadyen is ready for the launch of Project One Million.<br />JEFF GARD/Northumberland Today

Andrew McFadyen is ready for the launch of Project One Million. JEFF GARD/Northumberland TodayPrint

CAMPBELLFORD – For a Campbellford father, this is just another fight for funding, albeit with a different province.

Andrew McFadyen is lobbying Alberta Health to approve life-sustaining treatment for three-year-old Aleena Sadownyk, who was recently diagnosed with the rare enzyme deficiency MPS VI (Maroteaux-Lamy Syndrome). Those affected lack an enzyme in their blood that breaks down cellular waste in the body called glycosaminoglycan (GAG), which builds up in the bones, tissues, organs and muscles. It can lead to devastating symptoms such as heart and airway disease, corneal clouding, stiffening of the joints, shortened stature and premature death.

Treatment — an Enzyme Replacement Therapy called Naglazyme — can range from $300,000 for a small individual to $1 million for a young adult per year. There are currently nine children suffering from MPS VI in Canada and about 1,100 cases worldwide.

McFadyen and his wife Ellen’s nine-year-old son Isaac suffers from MPS VI, but he has been receiving treatment on a weekly basis for seven years at The Hospital for Sick Children in Toronto after he was original denied funding. The McFadyens lobbied and they won.

Since that time, the family has also successfully lobbied for funding for another Ontario boy, Jasper More, in 2011 and a Saskatchewan girl, Violet Revet, in 2012.

Now the focus is on the St. Albert, Alberta toddler Aleena Sadownyk, who was denied funding by Alberta Health through the Alberta Rare Diseases Funding Program. A second application through the Short Term Exceptional Drug Therapy program is currently being reviewed, McFadyen said.

“It’s awful… the fact this has to continue to play out the same way every time a child gets diagnosed in a new province,” McFadyen said, while noting it spotlights Canada’s need for an Orphan Drug Policy. “The little girl’s family has to wait (for the decision) and it’s not right. It’s tough reliving this experience, but you empathize with the family.”

His advocacy work includes checking in with Alberta Health several times per day, ensuring the ministry has received all of the information it needs to make a proper decision and know that precedent has been set. In addition, he works with opposition health critics and prepares news releases.

McFadyen said when a child was diagnosed with MPS VI in Quebec, that province followed precedent already set by Isaac and subsequent cases. He hopes Alberta will do the same.

These funding fights engender “mixed emotions” in McFadyen, who used the words “angry” and “stressful” to describe the process.

“This takes over everything in life but it’s a small sacrifice for a little girl and her family,” McFadyen said. “It’s rewarding when success comes, knowing what it means for the family and the kids.”

jeff.gard@sunmedia.ca

twitter.com/NT_jgard

06 Aug 2013
Sticky Post By Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

Toddler's family seeks relief from rare disease

Sticky Post By On 6 August, 2013

St. Albert child awaits approval of costly treatment

By: Stu Salkeld

|  Posted: Tuesday, Aug 06, 2013 12:15 pm

RARE CONDITION – Three-year-old Aleena Sadownyk of St. Albert is battling MPS VI, an extremely rare disease that governments seem to be slow to address.

RARE CONDITION – Three-year-old Aleena Sadownyk of St. Albert is battling MPS VI, an extremely rare disease that governments seem to be slow to address.
Supplied photo

The MLA for St. Albert said a very young member of his constituency is front and centre in the government’s, and public’s, eye.

Stephen Khan said he spent the weekend talking to people involved in the situation facing three-year-old Aleena Sadownyk. The three-year-old St. Albert resident has been diagnosed with the extremely rare Maroteaux-Lamy Syndrome, also called MPS VI.

Sufferers of MPS VI lack an enzyme in their blood that breaks down cellular waste in the body called glycosaminoglycan (GAG). These GAGs build up in the bones, tissues, organs, and muscles of affected individuals and lead to many devastating symptoms including heart and airway disease, corneal clouding, stiffening of the joints, shortened stature, and premature death. To date, there are nine children suffering from the disease in Canada and roughly 1,100 worldwide.

“This adorable little girl, there is a drug that helps treat the deficiency that she has,” said Khan from his home Monday.

“The catch is, the drug has not been approved for use in Canada.”

MPS VI is rare, and the treatment required to keep Aleena healthy is also rare and quite expensive, ranging from $300,000 to $1 million per year, but urgently needed to keep her situation from worsening. Naglazyme is the only treatment known for the condition, and Alberta Health Services has already denied funding under the Alberta Rare Diseases Funding Program, but Khan said other avenues are opening up.

“The family is under unimaginable strain and stress,” said Khan. He said his St. Albert office was notified July 4 about Aleena and her story was quickly forwarded to the minister of Health. From there, the minister’s office is to forward it to AHS.

He said, although Naglazyme is not approved for use in Canada, some provinces have made exceptions. Khan said he’s been talking with the ministry and also with representatives of the Sadownyk family to get the ball rolling more quickly. Khan said he wanted to meet directly with the Sadownyk family, but was unable to. However, the minister’s office stepped up and has been handling the situation.

“We’re hoping to get access as soon as possible because every day is an eternity for the family,” said Khan.

Frustration

Andrew McFadyen, whose son Isaac lives with MPS VI, lobbied the Ontario government to cover Naglazyme and has been instrumental in helping the Sadownyk family.

“I’ve just become more and more frustrated with what’s been transpiring,” said McFayden Tuesday.

“We’re essentially waiting on the province to make a decision and that’s why it’s important we do get an expedited decision on this matter. Every day that goes by is another day that she’s not receiving the therapy that she needs.

“We’re reaffirming our call for an immediate decision on this so that the family can know what the fate of their daughter is. The future of your child sits on the desk of a few bureaucrats that haven’t had the opportunity to work through a process they haven’t put in place in an expedited fashion and we’re just calling on them to do the right thing.”

McFayden knows this disease well as it afflicted his son. He knows what Aleena is facing.

“Already at three-and-a-half years old she’s suffering from a lot of the irreversible symptoms of this disease, her joints are starting to stiffen up, her hands are starting to claw up, she can’t raise her hands above her head,” said McFayden.

“Her energy level is severely, severely deteriorating, her internal organs are enlarged, there’s already evidence of buildup on the bones. And those are only the things that are prevalent. We just don’t know what sort of symptoms are just waiting on the doorstep to appear. None of these symptoms can be reversed, but as soon as treatment starts we hope that the majority of these symptoms will stop appearing.

“Her lifespan will be severely shortened without treatment.”

Khan said the issue is bringing people together, even in the legislature, as all parties seem to want to see an approval made for the treatment to help Aleena. “I think everybody in Alberta can be supportive of the Sadownyk family and their cause.”

Khan said everyone is waiting for the provincial committee that oversees the Short Term Exceptional Drug Therapy program to meet and make a decision. He said the federal government is also working on this issue through the Special Access Program, which already approved Naglazyme for MPS VI sufferers in Ontario, B.C., Saskatchewan, and Quebec. Naglazyme is already approved in other countries around the world, including the United States and members of the European Union.

Khan said no one knows what the committee is going to do yet, but he is optimistic.

“From the time our office first contacted the minister’s office, we’ve been getting regular updates,” said Khan.

“The last update we got was very positive, very hopeful. The quicker we can get a decision, the happier everyone is going to be.”
06 Aug 2013
Sticky Post By Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

Toddler’s family seeks relief from rare disease

Sticky Post By On 6 August, 2013

St. Albert child awaits approval of costly treatment

By: Stu Salkeld

|  Posted: Tuesday, Aug 06, 2013 12:15 pm

RARE CONDITION – Three-year-old Aleena Sadownyk of St. Albert is battling MPS VI, an extremely rare disease that governments seem to be slow to address.

RARE CONDITION – Three-year-old Aleena Sadownyk of St. Albert is battling MPS VI, an extremely rare disease that governments seem to be slow to address.
Supplied photo

The MLA for St. Albert said a very young member of his constituency is front and centre in the government’s, and public’s, eye.

Stephen Khan said he spent the weekend talking to people involved in the situation facing three-year-old Aleena Sadownyk. The three-year-old St. Albert resident has been diagnosed with the extremely rare Maroteaux-Lamy Syndrome, also called MPS VI.

Sufferers of MPS VI lack an enzyme in their blood that breaks down cellular waste in the body called glycosaminoglycan (GAG). These GAGs build up in the bones, tissues, organs, and muscles of affected individuals and lead to many devastating symptoms including heart and airway disease, corneal clouding, stiffening of the joints, shortened stature, and premature death. To date, there are nine children suffering from the disease in Canada and roughly 1,100 worldwide.

“This adorable little girl, there is a drug that helps treat the deficiency that she has,” said Khan from his home Monday.

“The catch is, the drug has not been approved for use in Canada.”

MPS VI is rare, and the treatment required to keep Aleena healthy is also rare and quite expensive, ranging from $300,000 to $1 million per year, but urgently needed to keep her situation from worsening. Naglazyme is the only treatment known for the condition, and Alberta Health Services has already denied funding under the Alberta Rare Diseases Funding Program, but Khan said other avenues are opening up.

“The family is under unimaginable strain and stress,” said Khan. He said his St. Albert office was notified July 4 about Aleena and her story was quickly forwarded to the minister of Health. From there, the minister’s office is to forward it to AHS.

He said, although Naglazyme is not approved for use in Canada, some provinces have made exceptions. Khan said he’s been talking with the ministry and also with representatives of the Sadownyk family to get the ball rolling more quickly. Khan said he wanted to meet directly with the Sadownyk family, but was unable to. However, the minister’s office stepped up and has been handling the situation.

“We’re hoping to get access as soon as possible because every day is an eternity for the family,” said Khan.

Frustration

Andrew McFadyen, whose son Isaac lives with MPS VI, lobbied the Ontario government to cover Naglazyme and has been instrumental in helping the Sadownyk family.

“I’ve just become more and more frustrated with what’s been transpiring,” said McFayden Tuesday.

“We’re essentially waiting on the province to make a decision and that’s why it’s important we do get an expedited decision on this matter. Every day that goes by is another day that she’s not receiving the therapy that she needs.

“We’re reaffirming our call for an immediate decision on this so that the family can know what the fate of their daughter is. The future of your child sits on the desk of a few bureaucrats that haven’t had the opportunity to work through a process they haven’t put in place in an expedited fashion and we’re just calling on them to do the right thing.”

McFayden knows this disease well as it afflicted his son. He knows what Aleena is facing.

“Already at three-and-a-half years old she’s suffering from a lot of the irreversible symptoms of this disease, her joints are starting to stiffen up, her hands are starting to claw up, she can’t raise her hands above her head,” said McFayden.

“Her energy level is severely, severely deteriorating, her internal organs are enlarged, there’s already evidence of buildup on the bones. And those are only the things that are prevalent. We just don’t know what sort of symptoms are just waiting on the doorstep to appear. None of these symptoms can be reversed, but as soon as treatment starts we hope that the majority of these symptoms will stop appearing.

“Her lifespan will be severely shortened without treatment.”

Khan said the issue is bringing people together, even in the legislature, as all parties seem to want to see an approval made for the treatment to help Aleena. “I think everybody in Alberta can be supportive of the Sadownyk family and their cause.”

Khan said everyone is waiting for the provincial committee that oversees the Short Term Exceptional Drug Therapy program to meet and make a decision. He said the federal government is also working on this issue through the Special Access Program, which already approved Naglazyme for MPS VI sufferers in Ontario, B.C., Saskatchewan, and Quebec. Naglazyme is already approved in other countries around the world, including the United States and members of the European Union.

Khan said no one knows what the committee is going to do yet, but he is optimistic.

“From the time our office first contacted the minister’s office, we’ve been getting regular updates,” said Khan.

“The last update we got was very positive, very hopeful. The quicker we can get a decision, the happier everyone is going to be.”
06 Aug 2013
Sticky Post By Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

PC MLA urges Alberta Health to move quickly to help toddler

Sticky Post By On 6 August, 2013

By   Global News

A St. Albert family is appealing to the Alberta Government to help save their daughter's life as she battles a rare disease, Friday, August, 2, 2013.

A St. Albert family is appealing to the Alberta Government to help save their daughter’s life as she battles a rare disease, Friday, August, 2, 2013. Supplied, The ISSAC Foundation

EDMONTON – The PC MLA for St. Albert, Stephen Khan, is urging Alberta Health to move quickly in making a “positive decision” for Aleena Sadownyk, a three-year-old who suffers a rare and debilitating disease.

The little girl was recently diagnosed with Matoeaux-Lamy Syndrome, also know as ‘MPS’.

Aleena lacks the enzyme needed to break down waste in her body, so it builds up on organs, bones, and muscles. The disease will cut her life short if she doesn’t get treatment.

However, the drug Aleena needs – Naglazyme – isn’t approved in Canada, so her family is appealing to the province to provide funding for the costly treatment.

In a message posted on his Facebook page Tuesday, Khan writes:

“As many of you have recently learned, a St. Albert family is seeking assistance from Alberta Health Services (AHS) to cover medication treatment for their three year old daughter’s rare medical condition. Aleena Sadownyk is an adorable three year old girl, who suffers from a potentially fatal and very rare enzyme deficiency, known as MPS VI. 

“MPS VI has proven to be treatable with a drug called Naglazyme. This drug, unfortunately, has not been approved for use in Canada. The Sadownyk family is currently seeking funding for the treatment from AHS, through the Province of Alberta’s Short Term Exceptional Drug Therapy Program.

“As the MLA for St. Albert, I fully endorse the Sadownyk’s request for funding support from AHS. My office and I have been advocating and working with the Minister of Health’s office on this issue since early July, when we first learned of the Sadownyk family’s situation.

“While I respect there is a process to follow for the Short Term Exceptional Drug Therapy Program review, I urge AHS to move quickly in rendering a positive decision for the Sadownyk family. Every day is an eternity for this young family, all the while knowing that a treatment for this debilitating disease is at hand. Currently four other provinces — Quebec, Ontario, Saskatchewan and British Columbia — have all made exceptions for families in need of this specific treatment.

“We remain hopeful that we will have a successful resolution for Aleena and her family. In the meantime, our thoughts and prayers go out to Aleena, her family, friends and supporters.”

The treatment costs $300,000 a year for a person Aleena’s age.

On Friday, Health Minister Fred Horne sent a statement to Global News:

“My heart goes out to the Sadownyk family. I know they want to do everything they can to help their daughter. The family has contacted my office recently and were immediately put in touch with appropriate officials who could assist in supporting the family in making a funding request through appropriate programs.

As things stand, Naglazyme has not yet received general market approval through Health Canada. Because it doesn’t have federal approval, the options for funding this drug are different than for some other drugs. Albertans who need access to high-cost drug therapies not already covered by provincial programs may explore funding options through the Short-term Exceptional Drug Therapy Program. That program relies on medical experts to assess whether a specific drug will be safe and effective for a specific patient. It’s done on a case-by-case basis, so it can take a patient’s needs and particular health condition into account.

“As a politician, I don’t get directly involved in assessing which drugs would be funded through that process – and appropriately leave that assessment to medical experts. Department officials will continue to support the application process, a clinical review is underway now and I understand we expect a response soon.”

Wildrose Health Critic Heather Forsyth has written a letter to the minister asking him to fund Aleena’s treatment.

“Every day this child waits her condition deteriorates. We need to help her,” says Forsyth.

 

06 Aug 2013
Sticky Post By Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

St. Albert MLA urges quick approval for child’s medical treatment

Sticky Post By On 6 August, 2013

August 6, 2013. 3:59 pm • Section: Capital Notebook, Uncategorized

Posted by:
Sarah O’Donnell

St. Albert’s Progressive Conservative MLA said Tuesday Alberta health officials should move quickly fund treatment for a three-year-old girl suffering from a rare medical condition.

Aleena Sadownyk’s family shared her story, and their push to get the provincial government to fund the expensive treatment that could keep the symptoms of Maroteaux-Lamy Syndrome at bay, last week. My Journal colleague Jodie Sinnema explained how Aleena is missing a crucial enzyme called glycosaminoglycan, which breaks down cellular waste in her body. Instead, the waste builds up, “restricting her movements, clouding her eyesight and damaging her organs. Without treatment, her life expectancy will dwindle.”

The St. Albert family has asked the province to approve funding for a drug called Naglazyme, which should help treat the condition.

On his Facebook page Tuesday, St. Albert MLA Stephen Khan said Alberta Health Services should approve funding through Alberta’s short term exceptional drug therapy program.

“As the MLA for St. Albert, I fully endorse the Sadownyk’s request for funding support from AHS,” Khan said. “My office and I have been advocating and working with the Minister of Health’s office on this issue since early July, when we first learned of the Sadownyk family’s situation.

“While I respect there is a process to follow for the Short Term Exceptional Drug Therapy Program review, I urge AHS to move quickly in rendering a positive decision for the Sadownyk family. Every day is an eternity for this young family, all the while knowing that a treatment for this debilitating disease is at hand. Currently four other provinces — Quebec, Ontario, Saskatchewan and British Columbia — have all made exceptions for families in need of this specific treatment.”

With his comments, Khan joins Wildrose health critic Heather Forsyth, MLA for Calgary-Fish Creek, who called on Health minister Fred Horne to immediately approve the funding for Aleena last Friday.

Alberta Health officials said Tuesday a clinical review by medical experts is underway. One of the issues is that Health Canada has yet to approve sale of the drug in Canada. “We’re not going to get involved to pressure them to rush that clinical review. We anticipate a response soon,” Alberta Health spokesman John Muir said Tuesday.

06 Aug 2013
Sticky Post By Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

Aleena Sadownyk, Alberta Girl With Rare Disease, Awaits Word On Treatment Funding

Sticky Post By On 6 August, 2013

Aleena Sadownyk, Alberta Girl With Rare Disease, Awaits Word On Treatment Funding

CP  |  By Dean Bennett, The Canadian PressPosted: 08/06/2013 5:19 pm EDT  |  Updated: 08/06/2013 9:31 pm EDT

Aleena Sadownyk

EDMONTON – A government member of the Alberta legislature added his voice Tuesday to the demand for immediate action to save a young girl wasting away due to a rare enzyme deficiency.

Stephen Khan, in a message posted on Facebook, wants Alberta Health Services to fast-track a decision on whether it will fund treatment for the disorder, which causes cellular waste to build up in the body. Khan is the Progressive Conservative member for St. Albert, where three-year-old Aleena Sadownyk lives.

“I urge AHS to move quickly in rendering a positive decision for the Sadownyk family,” wrote Khan.

“Every day is an eternity for this young family, all the while knowing that a treatment for this debilitating disease is at hand.”

Sadownyk has cellular waste building up in her joints and around her heart. A drug called Naglazyme can arrest the build up, but can’t do much for the damage already done.

The drug costs at least $300,000 a year to administer and has yet to be approved for use in Canada. It has been approved in numerous other countries including the United States.

Seven other Canadian children are receiving the synthetic enzyme through special exemptions funded by provincial governments in B.C., Ontario, Quebec, and Saskatchewan.

Khan said he has been working with health officials for a month trying to get the drug approved under the Short Term Exceptional Drug Therapy program.

Under the short-term program, the enzyme replacement would be provided for six months.

Health Department spokesman John Muir said Tuesday that an arm’s-length panel of medical experts is looking at the application.

He said the panel wants to move carefully given the drug has not been approved for use in Canada.

“We don’t want to be in a position where anyone is rushing a clinical review. We want to ensure that this drug is most appropriate for an individual and it’s done on a case-by-case basis,” said Muir.

He said the government received the request in mid-July and that the reviews generally take a few weeks to complete.

He said about 1,000 applications are made a year under the program and that 94 per cent are approved.

Sadownyk has already been denied funding for the treatment under the Alberta Rare Diseases Funding Program.

The Wildrose party wrote to Health Minister Fred Horne last week, criticizing the delays, urging he intervene personally to OK the treatment for Sadownyk.

“There have been major barriers thrown up in accessing this drug for Aleena, and each day of delay provides another opportunity for the irreversible symptoms of the disease to progress,” Wildrose critic Heather Forsyth said in a correspondence to Horne.

Muir said it would be improper for Horne to intervene.

“You don’t want to make it about politics at all. You want to leave those medical decisions to clinicians who have that expertise,” he said.

He said the Sadownyk family was denied funding under the rare diseases program because the drug didn’t qualify as it had not received general market approval through Health Canada.

The disease is called MPS VI, or Maroteaux-Lamy syndrome. Patients lack the enzyme in blood that breaks down cellular waste. The waste then accumulates in the bones, tissues, and organs, leading to stiffened joints, heart and airway blockages, and potential death.

Nine children have the disease in Canada.

06 Aug 2013
Sticky Post By Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

Pressure ramps up on Alberta to fund care for girl with rare disease

Sticky Post By On 6 August, 2013

DEAN BENNETT – GLOBE AND MAIL

EDMONTON — The Canadian Press

Published 

A government member of the Alberta legislature added his voice Tuesday to the demand for immediate action to save a young girl wasting away due to a rare enzyme deficiency.

Stephen Khan, in a message posted on Facebook, wants Alberta Health Services to fast-track a decision on whether it will fund treatment for the disorder, which causes cellular waste to build up in the body. Khan is the Progressive Conservative member for St. Albert, where three-year-old Aleena Sadownyk lives.

“I urge AHS to move quickly in rendering a positive decision for the Sadownyk family,” wrote Khan.

“Every day is an eternity for this young family, all the while knowing that a treatment for this debilitating disease is at hand.”

Sadownyk has cellular waste building up in her joints and around her heart. A drug called Naglazyme can arrest the build up, but can’t do much for the damage already done.

The drug costs at least $300,000 a year to administer and has yet to be approved for use in Canada. It has been approved in numerous other countries including the United States.

Seven other Canadian children are receiving the synthetic enzyme through special exemptions funded by provincial governments in B.C., Ontario, Quebec, and Saskatchewan.

Khan said he has been working with health officials for a month trying to get the drug approved under the Short Term Exceptional Drug Therapy program.

Under the short-term program, the enzyme replacement would be provided for six months.

Health Department spokesman John Muir said Tuesday that an arm’s-length panel of medical experts is looking at the application.

He said the panel wants to move carefully given the drug has not been approved for use in Canada.

“We don’t want to be in a position where anyone is rushing a clinical review. We want to ensure that this drug is most appropriate for an individual and it’s done on a case-by-case basis,” said Muir.

He said the government received the request in mid-July and that the reviews generally take a few weeks to complete.

He said about 1,000 applications are made a year under the program and that 94 per cent are approved.

Sadownyk has already been denied funding for the treatment under the Alberta Rare Diseases Funding Program.

The Wildrose Party wrote to Health Minister Fred Horne last week, criticizing the delays, urging he intervene personally to approve the treatment for Sadownyk.

“There have been major barriers thrown up in accessing this drug for Aleena, and each day of delay provides another opportunity for the irreversible symptoms of the disease to progress,” Wildrose critic Heather Forsyth said in a correspondence to Horne.

Muir said it would be improper for Horne to intervene.

“You don’t want to make it about politics at all. You want to leave those medical decisions to clinicians who have that expertise,” he said.

He said the Sadownyk family was denied funding under the rare diseases program because the drug didn’t qualify as it had not received general market approval through Health Canada.

The disease is called MPS VI, or Maroteaux-Lamy syndrome. Patients lack the enzyme in blood that breaks down cellular waste. The waste then accumulates in the bones, tissues, and organs, leading to stiffened joints, heart and airway blockages, and potential death.

Nine children have the disease in Canada.

06 Aug 2013
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Alberta girl with rare disease awaits word on funding from province

Sticky Post By On 6 August, 2013

HAMILTON SPECTATOR – AUGUST 6, 2013

EDMONTON A member of the Alberta legislature is adding his voice to the demand for immediate action to save a young girl wasting away due to a rare enzyme deficiency. Stephen Khan, in a letter on Facebook, wants Alberta Health Services to fast-track a decision on whether it will fund treatment for the disorder, which causes cellular waste to build up in the body.

Khan is the Progressive Conservative member for St. Albert, where three-year-old Aleena Sadownyk lives.

Sadownyk has cellular waste building up in joints and around her heart.

A drug called Naglazyme can stop further waste from building up, but can’t fix the damage already done.

The drug costs at least $300,000 a year to administer and provincial health officials are still deciding whether to approve it.

The Canadian Press

05 Aug 2013
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Family fights for funding to treat daughter’s rare condition

Sticky Post By On 5 August, 2013

August 5, 2013 by 

A St. Albert couple is waging a fight with the Alberta government for their daughter’s life.

Laura and Dane Sadownyk live in Erin Ridge with their three-year-old daughter Aleena who, earlier this year, was diagnosed with an extremely rare condition called MPS VI, or Maroteaux-Lamy Syndrome. Treatment is available, but could cost anywhere from $300,000 to $1 million a year, and the family is working with a foundation out of Ontario to get the province to fund treatment through its Short-term Exceptional Drug Therapy Program.

St. Albert

The parents of three-year-old Aleena Sadownyk are fighting to have treatment for their daughter’s rare medical condition funded by the Alberta government. (Photo supplied)

“It’s devastating to hear your child has any kind of condition that not only is rare, but takes time to find,” Dane said. “We’ve been through a wide range of emotions, for sure, as parents. You’re trying to absorb it all; it’s surreal at times.”

Currently, there are only nine cases of MPS VI in Canada and 1,100 worldwide. Sufferers lack an enzyme in their blood that breaks down cellular waste called glycosaminoglycan (GAG). This waste then builds up in bones, tissues, organs and muscles, causing heart and lung disease, stiffened joints, shortened stature and premature death.

Aleena was diagnosed with MPS VI in April, and since then, Dane said that she has dealt with it pretty well.

“She does have a very keen sense of what’s going on around her,” he said. “When your daughter starts asking questions like, ‘Are we going to the doctor today?’ and things of that nature, you know they’re sensing there’s something going on.”

There is no cure for MPS VI, but it can be managed through an enzyme replacement therapy called Naglazyme, which infuses small doses of a synthetic version of the enzyme Aleena is lacking on a weekly basis.

However, given how expensive treatment is, the Sadownyks need help from the provincial government. They have already applied to the Alberta Rare Disease Funding Program, but were told that was not the right program because Naglazyme had not been given general market approval through Health Canada.

“My heart goes out to the Sadownyk family. I know they want to do everything they can to help their daughter,” Alberta Health Minister Fred Horne said in a statement emailed to the Leader.

Health Minister Fred Horne. (IAN KUCERAK, Sun Media News Services)

Health Minister Fred Horne. (Sun Media News Services)

“Because [Naglazyme] doesn’t have federal approval, the options for funding this drug are different than for some other drugs. … [The Short-term Exceptional Drug Therapy Program] relies on medical experts to assess whether a specific drug will be safe and effective for a specific patient. It’s done on a case-by-case basis, so it can take a patient’s needs and particular health condition into account.”

Helping the Sadownyks fight their battle is the Isaac Foundation out of Ontario, headed up by Andrew McFadyen, whose son Isaac was also diagnosed with MPS VI in 2006 when he was 18 months old. He has been receiving Naglazyme treatment for about seven years and is doing “extremely well,” his dad said, and that has inspired him to help other families get the treatment they need.

“When Isaac was diagnosed, we were told there was a treatment available, but we were told by our physician, ‘Don’t even think about it because you’ll never get it here in Canada,’” McFadyen said. “For us, the only thing we could do is fight for our child, so that’s what we did. We made sure we could bring it to Canada, then we made sure we could deal with the bureaucrats to get it funded in Canada. From that, there was precedent set.”

McFadyen has helped families fight for Naglazyme funding in British Columbia, Saskatachewan and Quebec. But dealing with the Alberta government has been frustrating.

“It just seems kind of silly that a group of bureaucrats are going to sit down in a room again and review the same data and make a decision on the fate of this little girl based on what they do or do not know about the drug,” he said.

While the Sadownyks would like to get Aleena started on Naglazyme as soon as possible and say it “means the world” to have someone like McFadyen on their side, there is no set timeline for a decision from the provincial government.

“As a politician, I don’t get directly involved in assessing which drugs would be funded through that process — and appropriately leave that assessment to medical experts,” Horne said in his statement. “Department officials will continue to support the application process, a clinical review is underway now and I understand we expect a response soon.”

Meanwhile, Dane is holding out hope that treatment can begin as soon as possible.

“We have more hope now than if we were trying to navigate this on our own,” he said. “Having said that, I’ll never feel completely satisfied as a parent until the day comes and we can take her in for those treatments.”

— GLENN COOK, St. Albert Leader

03 Aug 2013
Sticky Post By Posted in Advocacy, Fight For Aleena, Media, Television Permalink

Alberta family appeals to province to save daughter’s life

Sticky Post By On 3 August, 2013

By Slav Kornik  Global News


Edmonton – Three-year-old Aleena Sadownyk was recently diagnosed with a rare disease called Matoeaux-Lamy Syndrome, also know as ‘MPS’.Aleena lacks the enzyme needed to break down waste in her body, so it builds up on organs, bones, and muscles.

The disease will cut her life short if she doesn’t get treatment.

The drug Aleena needs isn’t approved in Canada, so her family is hoping the province will fund the costly treatment.

“It’s something that she deserves. As any other child should have, we want her to have as full a life as possible and every opportunity she should have,” says Aleena’s dad, Dane Sadownyk.

“Her heart valve is affected, her liver and spleen are enlarged and of course the deposits on the bones. It will result eventually in death,” says Aleena’s mom Laura Sadownyk.

Aleena’s parents have applied for help. The treatment costs $300,000 a year for a person Aleena’s age.

Patients in Ontario, Saskatchewan, BC and Quebec have secured provincial funding for the drug and they say it’s changed their lives.

Andrew Mcfayden’s son is one of the patients who received the drug in Ontario.

Mcfayden is now trying to do the same for the Sadownyks.

“It’s nerve-racking, it’s frustrating…if you think about it the fate of their daughter rests in the hands of a few bureaucrats at Alberta Health and I wouldn’t wish that on anybody,” says Mcfayden.

Wildrose Health Critic heather Forsyth has written a letter to the minister asking him to fund Aleena’s treatment.

“Every day this child waits her condition deteriorates. We need to help her,” says Forsyth.

Health Minister Fred Horne has sent a statement to Global News:

“My heart goes out to the Sadownyk family. I know they want to do everything they can to help their daughter. The family has contacted my office recently and were immediately put in touch with appropriate officials who could assist in supporting the family in making a funding request through appropriate programs.

As things stand, Naglazyme has not yet received general market approval through Health Canada. Because it doesn’t have federal approval, the options for funding this drug are different than for some other drugs. Albertans who need access to high-cost drug therapies not already covered by provincial programs may explore funding options through the Short-term Exceptional Drug Therapy Program. That program relies on medical experts to assess whether a specific drug will be safe and effective for a specific patient. It’s done on a case-by-case basis, so it can take a patient’s needs and particular health condition into account.

“As a politician, I don’t get directly involved in assessing which drugs would be funded through that process – and appropriately leave that assessment to medical experts. Department officials will continue to support the application process, a clinical review is underway now and I understand we expect a response soon.”

But soon can’t come soon enough for the Sadownyks.

“She’s a little girl. She didn’t ask for this. She didn’t deserve it. She should be given every opportunity like any child to have the best shot at life,” says Aleena’s dad.

With files from Vassy Kapelos 

© Shaw Media, 2013

03 Aug 2013
Sticky Post By Posted in Advocacy, Fight For Aleena, Media, Television Permalink

Alberta family appeals to province to save daughter’s life

Sticky Post By On 3 August, 2013

By Slav Kornik  Global News


Edmonton – Three-year-old Aleena Sadownyk was recently diagnosed with a rare disease called Matoeaux-Lamy Syndrome, also know as ‘MPS’.Aleena lacks the enzyme needed to break down waste in her body, so it builds up on organs, bones, and muscles.

The disease will cut her life short if she doesn’t get treatment.

The drug Aleena needs isn’t approved in Canada, so her family is hoping the province will fund the costly treatment.

“It’s something that she deserves. As any other child should have, we want her to have as full a life as possible and every opportunity she should have,” says Aleena’s dad, Dane Sadownyk.

“Her heart valve is affected, her liver and spleen are enlarged and of course the deposits on the bones. It will result eventually in death,” says Aleena’s mom Laura Sadownyk.

Aleena’s parents have applied for help. The treatment costs $300,000 a year for a person Aleena’s age.

Patients in Ontario, Saskatchewan, BC and Quebec have secured provincial funding for the drug and they say it’s changed their lives.

Andrew Mcfayden’s son is one of the patients who received the drug in Ontario.

Mcfayden is now trying to do the same for the Sadownyks.

“It’s nerve-racking, it’s frustrating…if you think about it the fate of their daughter rests in the hands of a few bureaucrats at Alberta Health and I wouldn’t wish that on anybody,” says Mcfayden.

Wildrose Health Critic heather Forsyth has written a letter to the minister asking him to fund Aleena’s treatment.

“Every day this child waits her condition deteriorates. We need to help her,” says Forsyth.

Health Minister Fred Horne has sent a statement to Global News:

“My heart goes out to the Sadownyk family. I know they want to do everything they can to help their daughter. The family has contacted my office recently and were immediately put in touch with appropriate officials who could assist in supporting the family in making a funding request through appropriate programs.

As things stand, Naglazyme has not yet received general market approval through Health Canada. Because it doesn’t have federal approval, the options for funding this drug are different than for some other drugs. Albertans who need access to high-cost drug therapies not already covered by provincial programs may explore funding options through the Short-term Exceptional Drug Therapy Program. That program relies on medical experts to assess whether a specific drug will be safe and effective for a specific patient. It’s done on a case-by-case basis, so it can take a patient’s needs and particular health condition into account.

“As a politician, I don’t get directly involved in assessing which drugs would be funded through that process – and appropriately leave that assessment to medical experts. Department officials will continue to support the application process, a clinical review is underway now and I understand we expect a response soon.”

But soon can’t come soon enough for the Sadownyks.

“She’s a little girl. She didn’t ask for this. She didn’t deserve it. She should be given every opportunity like any child to have the best shot at life,” says Aleena’s dad.

With files from Vassy Kapelos 

© Shaw Media, 2013