denied | The Isaac Foundation

13 Dec 2015

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

Mom pleads with Liberal government to allow potentially life-saving treatment for child

Sticky Post By mcfadyena On 13 December, 2015

CHRIS MORRIS Legislature Bureau

December 13, 2015

Carolle Mazerolle with her 10-year-old son Morgan Doucet. Doucet sufferers from a rare disease called Morquio Syndrome and is believed to be one of only 33 people in the country and the only known case in New Brunswick.

Photo: Jeremy Trevors/ Miramichi Leader

Carolle Mazerolle has been battling medical bureaucracies to get help for her son, Morgan, almost since he was born 10 years ago.

Now she has another fight on her hands, this time to get a new treatment that could finally help her beloved and brave little boy enjoy not only a pain-free life but also, possibly, a longer life.

“I want to give him this drug as quickly as possible,” Mazerolle said in an interview, sending a message directly to Health Minister Victor Boudreau.

“I want them (New Brunswick health officials) to make another decision on this. Morgan needs this right away. He gets worse by the minute and every moment he waits, his body is deteriorating. I don’t want to wait another year, not even another day. Please, just make the right decision.”

Mazerolle fights tears as she speaks. It has been a long, difficult time for Carolle, her partner Glen Doucet, Morgan, and other members of the family in Baie-Sainte-Anne as they cope with Morgan’s rare genetic condition called Morquio Syndrome.

The family finally felt hope last year after it was announced that Health Canada had approved a treatment, an enzyme replacement therapy, that has been shown to slow down or halt progression of the disease in patients.

Morgan is the only known person in New Brunswick with Morquio Syndrome and Carolle and Glen quickly applied to have the treatment covered for their son. After about a year, their request was denied in October, and no avenue for appeal was offered.

It has been a roller-coaster ride for the family.

“We all were all so hopeful when the treatment was approved,” Carolle said. “We thought everything is going to fall into place and Morgan will have a better life and live a lot longer. Then they refused us. I still have trouble believing it”

The treatment is expensive.

Andrew McFadyen, executive director of The Isaac Foundation, an Ontario-based advocacy organization that specializes in diseases like the one afflicting Morgan, says the list price for the enzyme replacement drug is about $300,000 per year.

However, McFadyen said a lower cost likely could be negotiated with the drug developer once Morgen gets the go-ahead for treatment from the province.

McFadyen was in Fredericton last week to meet with Boudreau and apply pressure to give Morgan a second chance at the treatment. He did not get the reassurances he was seeking, although he said Boudreau did agree to look at the case.

“New Brunswick health officials are telling this family to enter a long-term palliative approach to care for their child instead of ensuring that disease progression is halted now,” he said.

“They are allowing him to die instead of taking leadership and doing the responsible and ethical thing by providing him with the help he needs. It’s shameful, disgraceful.”

The Health Department says it is bound by privacy rules and cannot speak about Morgan’s case. Jake Stewart, opposition Tory MLA for Southwest Miramichi-Bay-du-Vin, has raised the issue in the legislature, appealing to the Liberal government to put politics aside and “just help this child.”

Carolle, Glen and McFadyen are planning to travel to Fredericton on Wednesday to press their case to have the treatment approved. Morgan may be there as well, although it is not certain.

Carolle said she knew something was wrong with Morgan shortly after he was born, although most children with the syndrome do not have it confirmed until four or five years of age.

Her baby seemed in pain right from the start and she remembers spending the first four months of his life sleeping on her back, holding him.

Carolle kept telling doctors something was wrong and they kept telling her he was just a bit slow and would be fine. Finally, at 15 months of age, a doctor in Saint John took x-rays and discovered bones in Morgan’s neck were broken.

Now in Grade 5, Morgan is fully developed intellectually and socially, and has lots of friends. But he has never walked, can stand only for a few seconds holding onto something, has almost constant headaches and is often sick to his stomach.

The syndrome has deformed his body, but not his spirit, Carolle says.

“This is a small community and everyone knows him,” she said. “The other kids are all great with him.”

McFadyen said sufferers of Morquio Syndrome lack an enzyme in their blood that breaks down cellular waste in the body. The waste builds up in the bones, tissues, organs, and muscles and leads to such symptoms as heart and airway disease, corneal clouding, stiffening of the joints, shortened stature, and premature death.

He said the treatment being denied to Morgan dramatically slows or halts the disease, improves pulmonary function, walking ability, and gives patients the chance at a normal life.

There are 33 cases of Morquio in Canada. The treatment currently is funded in Saskatchewan, Ontario and Quebec.

Source: https://www.telegraphjournal.com/telegraph-journal/story/45053995/mom-pleads-with-liberal

06 Oct 2015

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

Health Ministry denies funding for Sask. siblings with rare genetic disease

Sticky Post By mcfadyena On 6 October, 2015

Lasia Kretzel

October 5, 2015 – 5:20pm

Muhammad Akhter spent 12 hours trying to figure out how to tell his wife some terrible news.

The couple’s three children have been denied treatment coverage from the province for their rare genetic disease, but Health Minister Dustin Duncan said he will seek a second opinion.

Muhammad Abdullah, 12, Khadija Amir, 10 and Sara Amir, 8 have an enzyme-related disease called Morquio A Syndrome (also called MPS IV type A). Their bodies lack an enzyme in their blood that breaks down cellular waste in the body. Without treatment it can lead to multiple diseases and premature death. The children have already experienced growth problems and stiff joints. Two of the them already use wheelchairs.

There is no cure, but treatment with a synthetic enzyme called Vimizim could slow down the disease and potentially extend the life of the children. The treatment would cost hundreds of thousands of dollars a year for each child.

“I was not expecting to be denied from the (common drug review’s) side because this is the last resort. This is the last hope,” Akhter said. “It’s a feeling, it’s a pain from the inside. It’s pretty hard.”

The Ministry of Health sought consultation from experts outside the province and provided information from the family and their physicians. Health Minister Dustin Duncan said they were told the drug wouldn’t be as effective on the children because they were older than five years.

“The longer it takes for this drug to be administered, the disease progresses to a point where it just becomes a question of whether or not there is efficacy to administering this drug,” Duncan said. “You would like to be able to say yes to everybody, but you want to ensure that for the sustainability of the system that you’re using the health care dollars as wisely as possible.”

Morquio A Syndrome only affects an estimated 100 people in Canada right now, according to the group Morquio in Canada.

IS IS THE LAST HOPE.

The Isaac Foundation said another Saskatchewan child has already received funding for the treatment, and Duncan said he only knew of one child being approved for therapy in recent memory.

The family, with the support of the foundation, met with the minister Monday to plead him to reconsider.

Duncan said because another child was approved for the drug before and because he feels there is debate in the medical community about the effectiveness of the drug at certain ages, he would like to review the siblings’ case.

“I’m going to ask the ministry to consult further with some other out-of-province experts to give me a second opinion on this,” Duncan said. “There’s not a lot of options for these patients so we want to make sure we give a full look before we close the door.”

Akhter said the family is now waiting anxiously, adding they aren’t sure what they will do if the second answer is still no.

“It’s like night and night; no shining,” he said.

06 Oct 2015

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

SASKATOON FAMILY DENIED FUNDING FOR TREATMENT

Sticky Post By mcfadyena On 6 October, 2015

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.“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 father Amir Akhter. Story continues below

Rare genetic disease diagnosed in 3 kids from same Saskatoon family

The siblings all have the disease which occurs when people are missing a genetic enzyme. It’s twisting their joints, seriously impacting their vision and hearing and they can hardly walk or run.

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 Amir 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 Amir Akhter.

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.

SOURCE: http://or-politics.com/canadahealth/saskatoon-family-denied-funding-for-treatment/102383/

06 Oct 2015

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

Funding For Rare Disease Treatment Denied, But Health Ministry Will Reconsider

Sticky Post By mcfadyena On 6 October, 2015

Written by Carol Thomson

Category: Local News

Created: 05 October 2015

The executive director of an advocacy group for rare diseases like Morquio Syndrome is puzzled as to why access for treatment has been denied for 3 Saskatoon siblings.

Andrew McFadyen, from the Isaac Foundation, along with the children and their father Amir Akhter spoke with Saskatchewan’s Health Minister this morning (Mon) in hopes of changing his mind.

McFadyen says the file on this case was sent to Ontario for review by only one person with no opportunity for different points of view, so today, he took the opportunity to speak to Dustin Duncan about why he should reverse the decision.

He adds that the Akhter family first requested access to provincial funding in March of 2014 and although the treatment wouldn’t cure the children, it could dramatically halt progression of the disease.

Dustin Duncan has said he will have another look at the case, but no date has been set for a decision.

The treatment is expensive at approximately $300,000 per year per child, but McFadyen says you could also argue that the cost of dealing with the effects of the disease when not treated could be similar when considering hip surgeries, corneal replacements, medical appointments, and other costs.

Duncan says the treatment costs $500,000 per patient.

There is a 2 year old in the province that has funding for treatment, which Duncan says is because the belief is that it’s more effective for children under 5.

McFadyen disputes that saying there is no data that actually proves that to be true.

SOURCE: http://www.saskatoonhomepage.ca/index.php?option=com_content&view=article&id=69454:funding-for-rare-disease-treatment-denied-but-health-ministry-will-reconsider&catid=61&Itemid=179

06 Oct 2015

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

Sask Health Minister wants second opinion on treatment for three Saskatoon children

Sticky Post By mcfadyena On 6 October, 2015

Saskatchewan Health Minister Dustin Duncan is asking for a second opinion on an unproven drug to treat three young Saskatoon children.

Initially coverage for the drug was denied last week.

The drug is been used to treat a very rare and fatal blood disease called Morquio Syndrome.

Health Minister Duncan says he wants to make sure they do their due diligence.

There is one child in the province who is on the drug to treat the same disease. However studies seem to show it’s effective for those under the age of five.

But in this case the three children are eight or older.

The drug costs five hundred thousand dollars a year per patient.

SOURCE: http://www.620ckrm.com/ckrm-on-air/ckrm-local-news/12882-sask-health-minister-wants-second-opinion-on-treatment-for-three-saskatoon-children

06 Oct 2015

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

Saskatchewan health minister to seek second opinion for family denied drugs

Sticky Post By mcfadyena On 6 October, 2015

at 18:36 on October 05, 2015, EDT.

The Canadian Press

REGINA – Saskatchewan’s health minister says he will ask for a second opinion on an unproven drug for three Saskatoon children with a rare and often fatal blood disease.

The Akhter family has met with Dustin Duncan to ask that the government cover the expensive treatment for Morquio syndrome.

It would cost $500,000 a year per child and the family’s funding request was turned down by ministry officials last week.

The drug is not a cure, although studies indicate it is effective in slowing down the disease in children under five.

In this case, the three children are eight, 10 and 12.

Duncan says he wants to make sure the department does its due diligence.

“It’s a very difficult situation, so I’m going to ask the ministry to consult further with some other out-of-province experts to give me a second opinion on this,” Duncan said Monday.

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

There is one child in the province who is on the drug.

Duncan said it’s important to get as much information as possible through the drug review process and from the manufacturers.

NDP Opposition critic Trent Wotherspoon said he feels the family should get coverage on compassionate grounds, even if the drug is not 100 per cent proven.

“They deserve a lifeline. They deserve some hope,” Wotherspoon said. “The potential of halting this degenerative disease and the progression of that disease is so critically important (to these children).”

Morquio syndrome is a hereditary disease in which the blood lacks a certain enzyme. The syndrome is characterized by skeletal defects such as stunted growth, deformity of the spine and chest, short neck and loose and enlarged joints. It can also lead to thin tooth enamel and corneal clouding.

The Isaac Foundation, an organization that funds research projects aimed at finding a cure for Morquio syndrome, is advocating for the Akhters.

Executive director Andrew McFadyen said the decision to deny funding was made on scant evidence.

“This was only looked at by one reviewer in Ontario, who clearly disregraded the international treatment guidelines, who clearly ignored the Canadian expert opinion on treating this disease,” McFadyen said.

The ministry has suggested the family take a long-term palliative approach to care for the children.

McFadyen suggested that may prove even more costly to the health-care system.

“When we look at the total cost of that drug, it’s often noted that the cost to not treat these children may be the same … when you look at interventions, medical appointments, hip replacements, corneal transplants, etc.”

(CKRM, CJWW, The Canadian Press)

Content Provided By Canadian Press.

Source: http://www.cfra.com/HealthCP/Article.aspx?id=481958

06 Oct 2015

Sticky Post By mcfadyena 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 mcfadyena 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.

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.

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

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.