Television

09 Oct 2015
Sticky Post By Posted in Advocacy, Blog Postings, Media, Morquio, MPS IVA, Television Permalink

Medical treatment funding plea for children with rare genetic disease

Sticky Post By On 9 October, 2015

David Giles, Senior Web ProducerBy Senior Web Producer  Global News

WATCH TV NEWS COVERAGE

SASKATOON – Amir Akhter is waiting for a life-changing phone call, a phone call that could determine the future of three of his children. Earlier this week, the Saskatchewan government denied funding an expensive treatment that may help them live longer.

The three children suffer from the same rare genetic disease.

“Skeletal disorder, hearing loss, vision loss, spinal cord problems, their heart, lungs, almost every part of the body,” said Akhter.

READ MORE: Rare genetic disease diagnosed in 3 kids from same Saskatoon family

But a treatment called Vimizim could change things. It costs hundreds of thousands of dollars and the Akhter’s were hoping the province could foot the bill.

That was rejected on Monday by the Saskatchewan government.

“The experts at this point have said there really doesn’t appear to be efficacy when it comes to halting the progression of the disease as they get older, but I want to have a second look at it,” Health Minister Dustin Duncan said at the time.

He added that because the kids are past the age of five, the treatment might not even work.

READ MORE: Treatment funding denied for 3 children with rare genetic disease

The Opposition NDP says that’s not true at all.

“That is the exact opposite of what the evidence says,” said Danielle Chartier, the NDP health critic.

“You can look at Vimizim’s own fact sheet that proves the drug is effective in those older than five.”

Chartier added that another child in the province is getting the same kind of treatment. Her age has not been confirmed.

For Akhter, it’s not about the back and forth disputes, it’s about watching his children deteriorate every single day.

“It’s heartbreaking, but we are not losing our heart, we are still very positive,” said Akhter.

“Hopefully, my kids will be getting that treatment.”

Biomarin Pharmaceuticals, a manufacturer of Vimizim, confirmed that safety and effectiveness have been proven for kids five year or older on their website.

The Health Canada website too says it is the other way around, that efficacy hasn’t been established for those under five.

The family is waiting to hear back about a clarification and a final decision.

Leena Latafat contributed to this story

06 Oct 2015
Sticky Post By Posted in Advocacy, Blog Postings, Media, Morquio, MPS IVA, Newspaper, Saskatoon Siblings, Television Permalink

Treatment funding denied for 3 children with rare genetic disease

Sticky Post By On 6 October, 2015

SASKATOON – It’s a parent’s worst nightmare; watching your child lose a battle to a devastating disease. Now imagine having to suffer through it multiple times.

Muhammad, Sara and Kadija Akhter suffer from a rare but crippling genetic disease called Morquio Syndrome that’s changed their childhood.

Video – http://globalnews.ca/video/2260432/treatment-funding-denied-for-3-children-with-rare-genetic-disease

“This syndrome is progressive. So every day they are losing something … they’re losing more and more. So it’s getting tougher for the family as well as the kids,” said their father, Amir Akhter.

There is no cure, but until recently, there was still hope. The family was anxiously hoping the province could pay for a treatment called Vimizim. The therapy could help them live longer and could even halt the progression of their disease.

The therapy costs hundreds of thousands of dollars.

On Monday, there was more heartbreak. The family was denied funding for the treatment.

“I should say that we are although walking and eating…we sometimes feel that we are not alive. And we have many challenges. And there is no hope from any end,” said Akhter.

It’s something Amir Akhter would never say in front of them but without therapy, Muhammad, Sara and Kadija could die. Still, they’re hopeful.

They spent Monday in Regina, urging the Saskatchewan Ministry of Health and the premier to reverse the province’s decision.

“Hopefully if we get that treatment … there is a new life, new whole life, new world, everything for us, for our family,” said Akhter.

READ MORE: Rare genetic disease diagnosed in 3 kids from same Saskatoon family

But Saskatchewan Health Minister Dustin Duncan argues that because the kids are past the age of five, this particular treatment may not even be effective.

“The health world is so complex and there are so many diseases and so many drugs. You want to be able to say yes to everyone but … healthcare dollars as wisely as possible,” Duncan said. Still, the minister says he will have a second look.

On the other hand, others argue that there’s no time for that and that this therapy is their last shot at survival.

“They deserve a lifeline. They deserve some hope. And the potential of halting this disease is so critically important to this eight-year-old, this 10-year-old this 13-year-old and of course, this family,” said Saskatchewan NDP deputy leader Trent Wotherspoon.

Although a decision has already been made, they are expected to hear back about a reversal in the next 10 days.

Until then, the family will hold on to all they have, their children and hope.

© Shaw Media, 2015

Source: http://globalnews.ca/news/2260420/saskatoon-family-denied-funding-for-treatment/

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

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

Parents want province to fund treatment for child’s rare condition

Sticky Post By On 3 August, 2013

Aleena Sadowynk, 3, has a rare enzyme deficiency CBC News

Posted: Aug 2, 2013 9:52 PM MT

Last Updated: Aug 2, 2013 9:50 PM MT


A couple from St. Albert is trying to get Alberta Health to cover the expensive medication to treat their daughter’s rare medical disorder.

Aleena Sadownyk, 3, suffers from the enzyme deficiency MPS VI, a condition with fewer than ten cases in Canada.

The MPS VI is treated with an drug called Naglazyme, which can cost anywhere from $300,000 to $1 million each year.

Aleena’s parents Dane and Laura Sadownyk applied for funding months ago but were turned down because because Health Canada hadn’t given Naglazyme general market approval.

They are now awaiting a reply from Alberta Health after applying under the Short Term Exceptional Drug Therapy Program.

“We’re not taking not taking no as an answer. If we have to contact every person out there to tweet, Facebook, email, phone, [Health] Minister [Fred] Horne, we’ll do that, ” Laura Sadownyk said.

“We have lots of people supporting us and we need the support of the public.”

“It’s a child’s life. That’s the bottom line,” Dane Sadownyk said. “She deserves a future like any other child should and that’s all we want for her.”

Aleena’s case is now being reviewed by medical experts who will determine whether the drug can be funded.

In a statement sent to CBC News, Horne suggested that the delay has been caused by the fact Naglazyme doesn’t have general market approval from Health Canada.

“Because it doesn’t have federal approval, the options for funding this drug are different than for some other drugs,” he said.

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

Parents want province to fund treatment for child's rare condition

Sticky Post By On 3 August, 2013

Aleena Sadowynk, 3, has a rare enzyme deficiency CBC News

Posted: Aug 2, 2013 9:52 PM MT

Last Updated: Aug 2, 2013 9:50 PM MT


A couple from St. Albert is trying to get Alberta Health to cover the expensive medication to treat their daughter’s rare medical disorder.

Aleena Sadownyk, 3, suffers from the enzyme deficiency MPS VI, a condition with fewer than ten cases in Canada.

The MPS VI is treated with an drug called Naglazyme, which can cost anywhere from $300,000 to $1 million each year.

Aleena’s parents Dane and Laura Sadownyk applied for funding months ago but were turned down because because Health Canada hadn’t given Naglazyme general market approval.

They are now awaiting a reply from Alberta Health after applying under the Short Term Exceptional Drug Therapy Program.

“We’re not taking not taking no as an answer. If we have to contact every person out there to tweet, Facebook, email, phone, [Health] Minister [Fred] Horne, we’ll do that, ” Laura Sadownyk said.

“We have lots of people supporting us and we need the support of the public.”

“It’s a child’s life. That’s the bottom line,” Dane Sadownyk said. “She deserves a future like any other child should and that’s all we want for her.”

Aleena’s case is now being reviewed by medical experts who will determine whether the drug can be funded.

In a statement sent to CBC News, Horne suggested that the delay has been caused by the fact Naglazyme doesn’t have general market approval from Health Canada.

“Because it doesn’t have federal approval, the options for funding this drug are different than for some other drugs,” he said.