06 Oct 2015

Sticky Post By mcfadyena Posted in Advocacy, Blog Postings, Media, Morquio, MPS IVA, Newspaper, Saskatoon Siblings Permalink

Province says no to medicine for three children, but will review decision

Sticky Post By mcfadyena On 6 October, 2015

Children suffering from crippling genetic disease
CBC News Posted: Oct 05, 2015 6:43 PM CT Last Updated: Oct 05, 2015 7:00 PM CT
The province’s health ministry says it won’t pay for a costly drug that may help three children with a crippling genetic disease, but Health Minister Dustin Duncan said he will ask for a second opinion.

Muhammad Abdullah, 12, Khadija Amir, 10 and Sara Amir, 8 all have a rare enzyme deficiency called Morquio A Syndrome (also called MPS IV type A).

Without the missing enzyme, cellular waste builds up in the bones, tissues, organs and muscles.

The condition is stunting the children’s growth, twisting their joints, affecting their eyesight and hearing, and making it tough for them to breathe. Two of them use wheelchairs at school.

Muhammad Amir Akhter is the children’s father. He said the disease can be life-threatening.

“Enzyme replacement therapy like Vimizim is the last resort,” Akhter said.

He said despite the current decision, he is staying positive. “It’s pretty hard for us, because this is the last hope here.”

Health minister will get a second opinion

Health Minister Dustin Duncan explained the decision to reject the drug coverage.

“These are very, very complex cases,” said Duncan.

He said in this case there is not enough evidence to prove that the drug will be effective for people over the age of five.

“There’s a lot of opinions on this. And so while we do rely and have relied on a previous case, on an out-of-province expert I’m asking the ministry to consult with others that may have some information on this particular case.”

Duncan said the ministry will review some of the medical debate about the efficacy of the drug, and will invite the Akhter family to submit any further evidence they find.

“While the answer last week was no, it doesn’t mean it’s no forever.”

The Opposition NDP said the children should get the drug since it is their only hope. It added that effectiveness can be monitored while they take it.

15 Sep 2015

Sticky Post By mcfadyena Posted in Advocacy, Blog Postings, Media, Morquio, MPS IVA, Newspaper, Saskatoon Siblings Permalink

Saskatoon family awaits answer on treatment for crippling disease

Sticky Post By mcfadyena On 15 September, 2015

Three children all suffer from genetic enzyme deficiency that could lead to early death
CBC News Posted: Sep 15, 2015 5:30 AM CT Last Updated: Sep 15, 2015 6:43 AM CT

A family in Saskatoon is waiting and hoping for the solution to a triple heartbreak. Three of the five children suffer from a crippling genetic disease that could kill them.

Muhammad Abdullah, 12, Khadija Amir, 10 and Sara Amir, 8 all have a rare enzyme deficiency called Morquio A Syndrome (also called MPS IV type A).

“Until the age of three to four nobody can judge that there’s anything wrong with them actually,” their father, Muhammad Amir Akhter said. “After that the symptoms appear, and almost every part of the body is affected.”

Without the missing enzyme, cellular waste builds up in the bones, tissues, organs and muscles.

The condition is stunting the children’s growth, twisting their joints, affecting their eyesight and hearing, and making it tough for them to breathe. Two of them use wheelchairs at school.

It’s so rare, it only appears in an estimated one out of every 200,000 to 300,000 children.

Hope placed in costly synthetic enzyme

There is no cure. But a synthetic enzyme called Vimizim could slow or even halt the progression of their disease, and help them live longer.

An advocacy group called the Isaac Foundation said Health Canada approved Vimizim more than a year ago.

Now Akhter and his wife Shazia Amir are waiting to find out if the province will pay the cost, which could be in the hundreds of thousands of dollars each year.

“It’s really hard to wait for the decision,” Akhter said. “You know we are anxiously looking forward to see every day morning, we are looking for any positive response from the government side to get that too, to get started that replacement therapy.”

Health ministry considering compassionate coverage

He said they applied seven months ago. However, Saskatchewan’s Ministry of Health said it only got complete information from the children’s doctor late last month.

It also said the national Common Drug Review made a “Do Not List” recommendation for Vimizim for clinical reasons.

However, the ministry is considering compassionate coverage on a case-by-case basis.

In the case of Akhter’s children, the ministry has sent their requests to an out-of-province specialist for review and advice.

If they are turned down, Akhter said he will consider moving the family to another province that is willing to pay for their treatment. The Isaac Foundation said Ontario and Quebec have reimbursed patients for Vimizim.

12 Aug 2013

Sticky Post By mcfadyena Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

Alberta will help fund critical treatment for young St. Albert girl

Sticky Post By mcfadyena On 12 August, 2013

BY SARAH O’DONNELL, EDMONTON JOURNAL AUGUST 13, 2013 7:34 AM

Alberta will help fund critical treatment for young St. Albert girl

Aleena Sadownyk has a rare enzyme deficiency called MPS VI that causes buildup of cellular waste in their body. They need a synthetic form of the enzyme to be injected each week.

Photograph by: Supplied , Edmonton Journal

EDMONTON – St. Albert father Dane Sadownyk picked up his three-year-old daughter Aleena and “just hugged her” Monday morning when the family learned the Alberta government will fund a crucial treatment for her rare medical condition.

“It was an extremely emotional moment,” Sadownyk said. “I was so elated. It felt like I could come up for a breath of air. That’s what it felt like, that I can breathe again.”

Aleena’s family and their supporters have been lobbying Alberta Health for a month to approve treatment for Maroteaux-Lamy Syndrome, a rare disease that means she lacks glycosaminoglycan, an enzyme that helps break down cellular waste.

Instead, the waste builds up, restricting movements, damaging organs and clouding eyesight, among other serious health complications. Without treatment, sufferers see their life expectancy cut short. Naglazyme, a synthetic enzyme approved in the U.S. but not Canada, can help break down that cellular buildup.

Though not a cure, weekly infusions could help prevent Aleena’s symptoms from getting worse, the family’s supporters say. Four other provinces have agreed to fund the treatment for seven children with the syndrome — also known as mucopolysaccharidosis type VI or MPS VI — and Aleena’s family was pushing Alberta to quickly approve the expensive but critical treatment, which is expected to initially cost about $300,000 a year.

Sadownyk, who was in Connecticut Monday with his family attending a conference on MPS VI when they heard the news, said they are feeling immense relief. Aleena was diagnosed with MPS VI in April.

“Today is definitely a day we are joyful for her and look forward to the future,” he said.

Andrew McFayden, director of the Isaac Foundation, went through a similar struggle in Ontario when his son Isaac was diagnosed with MPS VI as a toddler and was the first to receive treatment in Canada. He stepped in to assist the Sadownyks with their case and said he shared their feeling of elation Monday.

But McFayden also said the happiness is mixed with frustration that it took so long and that the family’s supporters felt they had to mount a public campaign with the support of MLAs such as Wildrose health critic Heather Forsyth, after the family’s original funding application through the Alberta Rare Disease Program was denied.

“We’ve gone through this numerous times,” McFayden said. “To me, that’s a big problem there still hasn’t been a process put in place by Health Canada for provinces to deal with funding these rare diseases.”

Health Minister Fred Horne said Monday he signed off on the funding for Aleena’s treatment through Alberta’s Short Term Evaluative Drug Therapy program, instead of the Rare Disease program, because Aleena’s case involves a drug not licensed for sale in Canada.

Horne said he weighed several factors, including the clinical evidence, the rarity of the disease, affordability of the drug and the best interest of the patient. Public pressure was not one of those factors, he said.

“I think Albertans would expect their minister and government to make these decisions based on evidence and looking at each case individually,” Horne said. “I’m pleased it’s able to be a positive outcome in this particular case. But these kinds of situations are becoming more common in Canada and it’s because we have more drugs coming out every day and more and more of these drugs are geared to rare diseases.”

Horne said he plans to talk about the need for an orphan drug program — a term used to describe medications for rare diseases — with other provincial health ministers and federal Health Minister Rona Ambrose.

“It’s an issue where we really need to collaborate,” Horne said. “There are only going to be more of these situations in the future.”

Forsyth, MLA for Calgary-Fish Creek, said Alberta Health must work to make the provincial system easier to navigate for families who suddenly find themselves seeking help with a rare condition. “The whole thing is just convoluted,” said Forsyth, who said she was overwhelmed to hear Aleena will receive treatment. “I think they have to simplify things and make it easier for the public to understand.”

NDP health critic Dave Eggen said Alberta Health also needs to speed up the process. “I’m glad something moved,” the Edmonton-Calder MLA said. “But in the future I don’t want to see people’s health compromised by being run through the wringer again.”

With Aleena’s funding approved, the Sadownyks’ next learning curve will be tied to her treatment.

“This is something that is new territory for us,” her father said. “We’ll learn.”

sodonnell@edmontonjournal.com

twitter.com/scodonnell

12 Aug 2013

Sticky Post By mcfadyena Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

Aleena Sadownyk will have treatment covered by Alberta government

Sticky Post By mcfadyena On 12 August, 2013

Little girl with rare disease had been rejected for funding previously

Beacon Reporter AB | August 12, 2013

The Alberta government has agreed to pay for the treatment of Aleena Sadownyk, who suffers from a rare disease.

After a month of impassioned pleas and lobbying the provincial government, Alberta Health will fund the necessary medical treatment for Aleena Sadownyk – a St. Albert girl with a rare medical condition.

Three-year-old Sadownyk has Maroteaux-Lamy Syndrome, a rare disease that causes cellular waste to build up in her joints and around her heart, restricting movements and damaging organs. A drug called Naglazyme will help, but while it is used in the United Sates, it has not been approved in Canada.

Early estimates indicated it could cost anywhere from $300,000 to $1 million a year to administer the drug, which Aleena Sadownyk would need to help fight the disease. Sadownyk’s parents had been pushing the government to pay for the treatment, along with dozens of others including opposition parties who joined the fight.

On Monday morning, Sadownyk’s father Dane said that the province had agreed to fund the treatment.

“The past few weeks have been very difficult for us, but we can now focus on improving Aleena’s well-being and look forward to her having a bright future ahead,” he said in a statement released by the Isaac Foundation – a patient advocacy group.

Aleena Sadownyk had been denied funding originally through the Alberta Rare Diseases Funding program back in July. During the last two weeks, all of Alberta’s opposition parties called on Health Minister Fred Horne to authorize the treatment.

“It is with tremendous joy that I thank all those who pushed so hard for the government to approve this life-saving treatment for Aleena,” said Wildrose health critic Heather Forsyth in a statement Monday morning.

“I hope that this painful ordeal for the Sadownyk family will lead to better approval processes for rare disease treatment in our province. We owe it to all Albertans to make sure that their health care system is there for them when rare diseases strike and extremely expensive treatments are their only hope.”

A spokesman with Alberta Health confirmed late Monday that the province will be paying for the drug to treat Sadownyk. The province was waiting for a clinical review to be completed on the benefits of Naglazyme before agreeing to fund it, said John Muir.

“We’ve come back now and carefully considered the circumstances and looked at that clinical review and we will now be funding Naglazyme for this individual,” he said. “We don’t want to be in a situation where we’re rushing any type of medical review on it. We want to make sure patient safety is put first and foremost and ensure it’s the best option for any individual.”

It has been reported that nine children in Canada are currently afflicted by Maroteaux-Lamy Syndrome. The cost of covering the Nagalyzme for Aleena Sadownyk will be around $300,000 a year.

12 Aug 2013

Sticky Post By mcfadyena Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

Aleena Sadownyk Treatment: Alberta To Pay For Drugs For 3-Year-Old With Rare Enzyme Deficiency

Sticky Post By mcfadyena On 12 August, 2013

CP | By Dean Bennett, The Canadian PressPosted: 08/12/2013 12:28 pm EDT | Updated: 08/12/2013 5:31 pm EDT

EDMONTON – The family of a three-year-old Alberta girl learned Monday she will receive a potentially life-saving drug for a disease that is causing cellular waste to build up in her joints and around her heart.

The province announced it will fund enzyme replacement therapy for Aleena Sadownyk of St. Albert, just outside Edmonton.

“It was very emotional,” Aleena’s father, Dane Sadownyk, said in an interview. “The first thing I did was pick up my daughter and give her a huge hug.

“It’s been a challenging and a tough road.”

A panel of medical experts with Alberta Health Services made the decision to fund the drug Naglazyme for Aleena.

Health Minister Fred Horne did not intervene in the decision, saying it needed to be made for medical reasons alone, but agreed it was a good day for the Sadownyk family.

“Obviously for the family it’s a positive outcome,” said Horne.

“These decisions are difficult, and we face more and more of them all the time in Canada as we have more drugs becoming available to treat rare … diseases.”

The drug costs $300,000 or more per year for children, and because the dosage is tied to weight, can rise to $1 million a year for adults. Those on it are on it for life as the drug does not cure the illness, but simply stops it from worsening.

The Sadownyks had been working with the province since the spring, after Aleena was diagnosed with Maroteaux-Lamy syndrome, also known as MPS VI.

MPS VI 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.

Dane said he and his wife, Laura, noticed something was wrong with Aleena — their middle child of three children — when she had trouble raising her arms, touching her shoulder or making a fist.

“We initially just thought she had arthritis because it does run in our family,” he said.

As they learned more about the disease they got in touch with Andrew McFadyen, who advocates for families dealing with MPS VI, to get funding for Naglazyme, which acts as an artificial enzyme to break down the cellular waste.

The drug is not approved yet for use in Canada, although it is in other countries such as the United States. It is permitted in special cases in Canada and is paid for in B.C., Saskatchewan, Ontario, and Quebec.

Of the nine children in Canada with the illness, Aleena was the only one not getting the drug prior to Monday, said McFadyen.

Aleena was initially denied funding for the treatment under the Alberta Rare Diseases Funding Program, but was approved Monday under the Short Term Exceptional Drug Therapy program, which provides the treatment for six months.

Horne said it’s up to the doctors to decide how and when the drug is administered after that.

“The medical experts will make decisions around how the drug is made available, how the monitoring takes places, and with respect to the ongoing coverage,” he said.

The parties had been quietly working on Aleena’s case for weeks when McFadyen, with the help of Heather Forsyth, health critic for official opposition Wildrose party, went public 10 days ago with a plea to Horne to intervene.

“I was hitting roadblock after roadblock (along with) misinformation and a lack of knowledge about the process,” said McFadyen.

“We couldn’t just leave it in the hands of the minister’s office to work through quietly. So that’s why we went public.”

Aleena’s plight took off on social media and the opposition parties, even St. Albert government member Stephen Khan, publicly pushed for an expedited decision.

Forsyth said she was overwhelmed Monday.

“The prognosis wasn’t good if she didn’t get it,” said Forsyth. “We’ve just given this little girl a whole new lease on life, and jeepers why didn’t the government do something about this (sooner).”

Horne said the criticism that the government moved slowly is unfair. He said those who had to make the decision were dealing with an unapproved drug and had to make sure it was right for this patient.

“I think the decision was made on a timely basis,” he said.

08 Aug 2013

Sticky Post By mcfadyena Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

Editorial: Life-saving treatment a worthwhile investment

Sticky Post By mcfadyena On 8 August, 2013

August 8, 2013 by editor

What’s good for the goose, they say, is good for the gander.

That’s all fine and dandy for birds who fly south in the winter, but is what’s good for the Ontarian good for the Albertan? What about what’s good for the Quebecer?

When it comes to treatment for the life-threatening medical condition known as MPS VI, it certainly is.

This extremely rare condition — there are currently only nine cases reported in Canada, and 1,100 worldwide — came into the limelight earlier this week when we found out that a three-year-old girl from

St. Albert, Aleena Sadownyk, was diagnosed with MPS VI earlier this year and that her family is wrestling with the Alberta government to have the treatment that could save her life funded through health care programs.

The drug she needs has not been fully approved by Health Canada, meaning that the Alberta government is dragging its feet by conducting its own clinical review. But similar reviews have already been conducted by governments in Ontario, Quebec, British Columbia and Saskatchewan, where funding for MPS VI treatment is already in place, and there is very little chance Alberta is going to find anything different. So why not get the funding in place already? There is ample evidence to prove that it works and is a worthwhile investment, even at $300,000 to $1 million per year.

And kudos to St. Albert MLA Stephen Khan for speaking out on Tuesday and calling for the treatment to be funded sooner rather than later. It’s refreshing to hear an MLA remember that his first job is to represent and advocate for the people of his riding, rather than to simply toe the Progressive Conservative party line.

Every day that passes puts Aleena Sadownyk’s young life further in jeopardy. The least the Alberta government — and especially Health Minister Fred Horne — could do is stop dragging its feet and take action.

— GLENN COOK, St. Albert Leader

07 Aug 2013

Sticky Post By mcfadyena Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

Local man fights for Alberta child’s health

Sticky Post By mcfadyena On 7 August, 2013

By Jeff Gard, Northumberland Today

Wednesday, August 7, 2013 1:43:27 EDT PM

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 mcfadyena Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

Toddler's family seeks relief from rare disease

Sticky Post By mcfadyena 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.

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 mcfadyena Posted in Advocacy, Fight For Aleena, Media, Newspaper Permalink

Toddler’s family seeks relief from rare disease