17 Dec 2015

Sticky Post By mcfadyena Posted in Advocacy, Media, Morquio, MPS IVA Permalink

HEALTH MINISTER REFUSES TO HEAR EXPERT OPINION REGARDING DENIAL OF LIFE-SAVING MEDICATION FOR MORGAN DOUCET

Sticky Post By mcfadyena On 17 December, 2015

Minister Refuses To Allow Renowned MPS Expert To Join Family Meeting; Doucet’s Family In Fredericton to Meet with Child And Youth Advocate Thursday

The family of ailing 10-year-old Morgan Doucet has cancelled a scheduled meeting with the Minister of Health in New Brunswick after the Minister refused to allow an internationally renowned expert to participate in the discussions. Doucet’s case has made headlines throughout the Province after the Minister of Health denied access to the life-saving drug that he requires. Morgan suffers from MPS IVA (Morquio Syndrome) and requires the life-saving treatment immediately in order to halt further progression of his devastating disease. Without access to therapy, Morgan faces a long and painful decline and death at a young age.

Andrew McFadyen, Executive Director of The Isaac Foundation, an advocacy, research, and family support organization that specializes in MPS related diseases, has slammed the Minister for his refusal to meet with one of the world’s foremost experts in MPS and related diseases. “It’s unconscionable to me that Minister Boudreau would refuse to allow this expert to participate in the meeting he had scheduled with Morgan’s family. Unbelievable, really. He has denied Morgan access to the life-saving medication he needs, hasn’t allowed his family an avenue to appeal his ruling, and then refuses to hear from a true expert that deals with this disease on a daily basis – an expert who is internationally recognized as the best in his field. It leads me to believe that he’s afraid to be proven wrong on such an important issue, which is deplorable because a life hangs in the balance. Pride and politics should never play a role in the health and well being of a child, and it appears that’s exactly what is happening with Morgan Doucet with respect to the Minister’s steadfast refusal to hear more evidence on this matter.”

Doucet’s parents will be joined McFadyen at the provincial legislature in Fredericton on Thursday, December 17 to watch the morning legislative session and to meet with the province’s Child and Youth Advocate, Norman Bosse. PC Jake Stewart, the Doucets’ MLA, will join the family during that meeting.

Bosse has agreed to look into the process used to deny access to Morgan’s medication, a process that McFadyen says was destined to return a negative decision all along. He notes that after an initial application for treatment in October of 2014, the file was finally sent to Ontario for review by one individual alone, with no opportunity allowed for a rigorous discussion with the rest of the MPS Experts throughout Canada and Internationally –experts that deal with the disease on a daily basis and some of whom have seen first-hand how well other children receiving the treatment are doing.

“The Minister and the Ministry’s only comment on this case is that the drug didn’t get a positive review from the Common Drug Review (CDR). What he doesn’t say is that all drugs used to treat other MPS diseases have received negative CDR reviews and the Province pays for access to all. Right now, Morgan is the only MPS patient in the province who has been denied this coverage, and that’s disgraceful.”

Health Canada approved the treatment Morgan requires in July of 2014 and it has been recommended for use in patients by the Canadian Expert Opinion on Morquio Syndrome. In addition, the International Expert Opinion on Morquio Syndrome considers the treatment their gold standard of care for patients throughout the world. It is reimbursed for patients in Saskatchewan, Ontario, and Quebec.

In Canada, there are 33 patients receiving the treatment. According to McFadyen, all of them are doing exceptionally well. “These patients have seen dramatic improvements and have had their disease progression either halted or dramatically slowed down. Morgan deserves the same chance at life that those other patients have. This is Canada – where equal access to our health care system isn’t a privilege, it’s a right.It’s callous and cruel that the Minister of Health is forcing this child and this family to enter a palliative approach to his disease when we know he can be helped. We know he can be saved.”

Jamie Myrah, Executive Director of the Canadian MPS Society, a national patient association that serves those affected by MPS and related lysosomal diseases, says Doucet’s case has angered and galvanized the people of New Brunswick. Doucet was front-page news on Monday, and has been featured by numerous provincial and national media outlets since his treatment was denied. The Isaac Foundation’s online posts about Morgan have received over 100,000 visitors during that time, and hundreds of emails have been sent to the Premier and the Minister of Health supporting access for Morgan. McFadyen has posted an online form that the people can fill out and all messages get delivered instantly to the Minister’s and the Premier’s email inbox.

“People in New Brunswick and across Canada are understandably upset about this decision and they want their voices heard,” says Myrah.

McFadyen finishes, “We won’t accept the Minister making medical decisions and overruling and contradicting medical experts, clinicians, and researchers regarding best practice and treatment for Morgan Doucet. If he won’t look at the clinical expert opinion, the patient reported outcomes or the peer-reviewed published journals, then who will he listen to?”

Both The Isaac Foundation and The MPS Society are renewing their call for Health Minister Victor Boudreau and Premier Brian Gallant to take action, review and reverse their initial decision, and to ensure that treatment begins immediately for Morgan Doucet.

# # #

The Doucet Family and The Isaac Foundation will be available for interested media at the Legislature on Thursday, December 17. To schedule an interview with Andrew McFadyen, please call Andrew at 613-328-9136 or email Andrew at mcfadyena@me.com. The Isaac Foundation can also arrange interviews with parents of patients currently receiving this treatment in Canada.

BACKROUNDER

While not a cure for Morquio Syndrome, the necessary Enzyme-Replacement Therapy (ERT) is designed to provide patients with a synthetic version of the enzyme they are lacking by infusing small doses into the patient’s bloodstream on a weekly basis. The treatment slows down or halts progression of the disease in patients, improves endurance, walking distance, breathing problems, and provides other benefits to sufferers that dramatically improve their quality and length of life. International experts and a Canadian Panel of Genetics Specialists have all recommended Vimizim as the front-line treatment for Morquio Syndrome. Vimizim was approved by Health Canada in July 2014, and is currently being reimbursed for use by patients in Saskatchewan, Ontario, and Quebec. Recently, the National Institute for

Photo Source: CBC.ca

09 Dec 2015

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

Baie Sainte Anne Boy Needs Drug To Manage Rare Disease

Sticky Post By mcfadyena On 9 December, 2015

December 9, 2015

A young Baie Sainte Anne boy’s plight made it to the floor of the provincial legislature this morning.

Morgan Doucet, 10, is currently the only person in New Brunswick suffering from morquio syndrome.

On Wednesday morning, Southwest Miramichi-Bay du Vin MLA Jake Stewart called on the province reverse its decision not to fund a Health Canada approved treatment for Doucet’s illness.

According to the U.S. National Library of Medicine, morquio syndrome is an inherited disease of metabolism in which the body is missing or doesn’t have enough of a substance needed to break down long chains of sugar molecules called glycosaminoglycans (formerly called mucopolysaccharides).

Symptoms include:

– Abnormal development of bones, including the spine
– Bell-shaped chest with ribs flared out at the bottom
– Coarse facial features
– Hypermobile joints
– Knock-knees
– Large head (macrocephaly)
-Short stature with a particularly short trunk
– Widely spaced teeth

While Stewart didn’t name the treatment in question, the only Health Canada approved drug for the disease is Vimizim, which was approved last year.

Annual treatment can cost upwards of $100,000.

Stewart said Doucet needs treatment.

“Recently access to this lifesaving medication for Morgan Doucet was denied by the minister of health and the department, even though it is the gold standard of care as recommended by the international treatment guidelines, has been recommended by the Canadian Expert Panel on Morquio Syndrome and has been prescribed by Morgan’s genetic specialist at the IWK ” Stewart said in the legislature.

He accused the government of overruling the experts and said there is no process for appeal.

Health Minister Victor Boudreau said he could not discuss the specifics of the case on the floor of the legislature, citing privacy rules, but said their is a process in place for situations like Doucet’s.

“Because we do not have the level of expertise on these rare diseases and rare drugs here in the province of New Brunswick we rely on the province of Ontario and this is a process that was put in place by the member for Rothesay, the former minister,” Boudreau said.

He explained that the file is sent to experts in Ontario and they get back to the New Brunswick government with a recommendation.
But Stewart wasn’t happy with that reply.

“The drug that Morgan requires is currently being funded by Saskatchewan, Ontario and Quebec and in most developed countries. Morgan’s application was denied by a single reviewer from Ontario, a reviewer who no longer practices medicine and has never used this treatment,” Stewart said.

He also said Saskatchewan found flaws with the review process and with that reviewer. He said that province now deals directly with the experts on specific diseases.

“I ask the minister of health and the premier, will they show the same leadership and do the right thing by ordering a new review of Morgan’s file and provide immediate access to this life saving treatment while that review is taking place?” Stewart asked.

Boudreau said these are very tough situations.

“Everybody in this legislature has a heart and understands that these decisions are difficult to make,” Boudreau said.

“I can tell you that across the country, and in New Brunswick, some cases get approved, some cases don’t. We follow a process, Mr. Speaker. I am not an expert, and we don’t even have those experts within our department or within our province,” he added, saying the process was followed.

09 Dec 2015

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

Health minister under pressure to approve expensive drug for sick boy

Sticky Post By mcfadyena On 9 December, 2015

CHRIS MORRIS Legislature Bureau

December 9, 2015

Andrew McFadyen, executive director of The Isaac Foundation, travelled from Ontario to Fredericton on Wednesday to lobby the Liberal government on behalf of Morgan Doucet, a 10-year-old boy from the Miramichi region, who has been denied treatment for a life threatening condition.

Photo: Chris Morris/Legislature Bureau

FREDERICTON • The New Brunswick Health Department is under pressure to reconsider the case of a little boy in northern New Brunswick who has been denied access to an expensive medical treatment that could improve and possibly lengthen his life.

Jake Stewart, opposition Tory MLA for Southwest Miramichi-Bay du Vin, raised the issue in the legislature on Wednesday, appealing to Health Minister Victor Boudreau to reconsider his department’s earlier decision to deny the child, Morgan Doucet of Baie-Sainte-Anne, a promising new treatment for the rare disease, Morquio Syndrome.

The treatment, which would cost about $300,000 a year for Morgan, has been approved by Health Canada but it was not given the go-ahead in New Brunswick following a review. The New Brunswick Health Department formally rejected the Doucet family’s application for help in October and has not provided an avenue for appeal.

Stewart said the family now has decided to allow their situation to become public. He said it is a heartbreaking case, made worse by what he described as a seemingly callous and bureaucratic response to the plight of a child in need.

“It is really simple what they (health officials) have to do: he is a 10-year-old boy; he is the only citizen of New Brunswick who has this disease; it is life-threatening – help him,” Stewart said in an interview.

“That is all we are asking. It’s not about politics – just help this little boy.”

Boudreau told the legislature that he cannot comment on the case specifically because of privacy issues. But he insisted he is not heartless.

“These are very difficult situations,” Boudreau said. “These are not easy files to deal with. I can assure you of that. Everybody in this legislature has a heart and understands that these decisions are difficult to make.

“Without talking about the case in particular, I can tell you that, across the country and in New Brunswick, some cases get approved and some cases do not. We follow a process.”

After question period on Wednesday, Boudreau met privately with Andrew McFadyen of the Ontario-based Isaac Foundation who travelled to Fredericton to press the minister on Morgan’s case.

“He made no commitments or promises,” McFadyen said of Boudreau. “He believes in the process currently in place but he will take a step back and look at this case and be in touch with me.

“I’ve been trying to talk with him about this decision since it was rendered in October and I have been stonewalled. It is encouraging but I had hoped for a bit more, at least a commitment to have a second review of this.”

The Health Department would neither confirm nor deny that the minister has agreed to take another look at the file, citing privacy concerns.

The process followed by the Health Department involved sending the file to an independent reviewer in Ontario who felt the best that could be expected from the costly treatment is “some slowing of the progression” of the disease.

McFadyen’s own son has a different form of Morquio Syndrome. It is believed there are only 33 cases in Canada, and Morgan Doucet is the only known case in New Brunswick.

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.

The treatment currently is funded in Saskatchewan, Ontario and Quebec.

Source: https://www.telegraphjournal.com/telegraph-journal/story/45005525/health-minister-under-pressure

03 Nov 2015

Sticky Post By mcfadyena Posted in Advocacy, Media, Saskatoon Siblings Permalink

LIFE-SAVING TREATMENT APPROVED FOR SASKATOON SIBLINGS

Sticky Post By mcfadyena On 3 November, 2015

Ministry of Health Reverses Funding Decision; Family Elated That “Hope Has Returned”

FOR IMMEDIATE RELEASE: The Minister of Health in Saskatchewan has approved funding for a life-saving treatment required by three siblings from Saskatoon. 8 year-old Sara Amir, along with her siblings Khadija, 10 and Muhammad, 12, suffer from MPS IVA (Morquio Syndrome) and require the life-saving treatment immediately in order to halt further progression of their devastating disease. After initially denying funding for the children, the Minister of Health ordered a review of the files and asked a panel of experts to provide feedback to the Ministry regarding the children and the treatment required.

Sufferers of Morquio Syndrome lack an enzyme in their blood that breaks down cellular waste in the body. This waste builds 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.

Andrew McFadyen, Executive Director of The Isaac Foundation, an advocacy, research, and family support organization that specializes in MPS related diseases, was thrilled with the news. “I applaud the Minister for his leadership during this review, and thank him for the open and transparent way with which he conducted this review. This is life-saving for these children, and life-changing for their family. This is incredible news.”

Jamie Myrah, Executive Director of The Canadian MPS Society, also thanked the NDP Health Critic, Danielle Chartier, for her work bringing this issue to the legislature and for the advocacy she provided the family during what was a very emotional period.

McFadyen adds, “The Saskatchewan government has taken a leadership role on this and have come in line with the Canadian Expert Opinion and the International Treatment Guidelines for Morquio Syndrome. They have also mirrored the recent recommendations from the UK. Most importantly, they’ve given hope back to this family – hope for a new life and the chance at fighting back against this relentless disease. For that, I am incredibly thankful to Minister Duncan and his team.”

# # #

For more information about this topic, or to schedule an interview with Andrew McFadyen, please call Andrew at 613-328-9136 or email Andrew at mcfadyena@me.com. The Isaac Foundation can also arrange interviews with parents of patients currently receiving this treatment in Canada.

Backgrounder

While not a cure for Morquio Syndrome, the necessary Enzyme-Replacement Therapy (ERT) is designed to provide patients with a synthetic version of the enzyme they are lacking by infusing small doses into the patient’s bloodstream on a weekly basis. The treatment slows down or halts the progression of the disease in patients, improves endurance, walking distance, breathing problems, and provides other benefits to sufferers that dramatically improve their quality and length of life. International experts and a Canadian Panel of Genetics Specialists have all recommended Vimizim as the front-line treatment for Morquio Syndrome. It was approved by Health Canada in July 2014, and has been reimbursed for use by patients in Saskatchewan, Ontario, and Quebec. Recently, the National Institute for Health and Care Excellence (NICE) recommended reimbursement for all 88 patients suffering from Morquio Syndrome throughout the UK.

09 Oct 2015

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

Medical treatment funding plea for children with rare genetic disease

Sticky Post By mcfadyena On 9 October, 2015

By David GilesSenior 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.

Those are the symptoms of Morquio syndrome, a degenerative enzyme disease. Three of Akhter’s children, Muhammad, Sara and Kadija, suffer from it. They’re missing an important enzyme and because of it, they could die.

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.

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

09 Oct 2015

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

NDP calls for reversal on denied drug coverage

Sticky Post By mcfadyena On 9 October, 2015

By François Biber

October 8, 2015 – 6:15pm

New Democratic Party MLA Danielle Chartier is challenging the province to reverse its decision to deny medical coverage for the children of a Saskatoon family with a rare disease known as Morquio A Syndrome.

“The Sask. Party government has denied three children their only hope to stop the progression of a terrible and debilitating disease it’s clear they have used incorrect information to make that decision,” Chartier told reporters in Saskatoon.

Earlier this week Muhammad-Amir Akhter was told by Health Minister Dustin Duncan, the province would not cover his children’s Vimizim treatment, a synthetic enzyme that helps the body break down cellular waste. Duncan added he was seeking out-of-province advice to see if this is the best treatment for Muhammad Abdullah, 12, Khadija Amir, 10 and Sara Amir, 8.

But Chartier said there are already 29 patients in Canada using the treatment, including one in Saskatchewan.

“First of all the government should be doing this on a compassionate basis, this is these children’s only hope, so if the government isn’t going to be compassionate they need to look at the evidence,” Chartier said, adding she believes Duncan’s claim that the drug hasn’t been proven to work for children under the age of five is completely false.

According to Health Canada, which has approved Vimizim, the safety and efficacy of the drug have not been established in children less than five years of age.

However, a common drug review conducted by the Canadian Agency for Drugs and Technologies and Health (CADTH) states in a report that evidence did not support the achievement of outcomes known to be clinically relevant to patients using Vimizim. The report also states the long-term safety profile of Vimizim requires further evaluation.

But for Akhter’s children, the situation isn’t getting any better without treatment.

“It’s heart-breaking. I understand, but we’re not losing our heart. We are staying positive and hopefully my kids will be getting the treatment and we will be able to see them happy and healthy in the future, I’m sure,” he said.

Coming in at around $300,000 per treatment, Akhter said they can’t afford treatment for three children without help from the province.

08 Oct 2015

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

Provincial opposition says drugs for sick kids denied over false information

Sticky Post By mcfadyena On 8 October, 2015

Expert says drug would work well for Akhter children

CBC News Posted: Oct 08, 2015 11:15 AM CT Last Updated: Oct 08, 2015 11:15 AM CT

Sara Amir, 8, Khadija Amir, 10, and Muhammad Abdullah, 12, all suffer from a crippling enzyme deficiency that threatens their lives. (Kathy Fitzpatrick/CBC)

CTV News – Critic calls for access to life-saving drug

Sticky Post By mcfadyena On 8 October, 2015

07 Oct 2015

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

Drug funding refusal for three children afflicted with rare disease to be reviewed

Sticky Post By mcfadyena On 7 October, 2015

Approval ‘the hope of our life’

BY JONATHAN CHARLTON, THE STARPHOENIX OCTOBER 6, 2015

Muhammad Amir Akhter helps his children, from left, Khadija, Sara and Muhammad Abdullah put their shoes on before heading to school at River Heights. The provincial health ministry is reconsidering drug funding for the children, who have an enzyme disorder.

Photograph by: Gord Waldner, The Starphoenix , The Starphoenix

The father of three children afflicted with a rare enzyme disease remains hopeful the province will pay for a costly drug therapy, despite receiving word last week that the Health Ministry had rejected his application.

Health Minister Dustin Duncan met Monday in Regina with Muhammed Akhter and his children and agreed to have the ministry seek a third opinion on whether Vimizim will be effective in treating their cases of a debilitating enzyme deficiency, Morquio syndrome or mucopolysaccaridosis IVA (MPS).

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

“Experts so far say there really isn’t efficacy when it comes to halting progression of the disease as children get older, but I want to have a second look at it.”

Akhter called the possibility of a positive result “the hope of our life.”

Vimizim, which can halt progression of the disease, can cost up to $460,000 per year.

Duncan said the children were denied approval based on the “Do not list” recommendation by the federal Common Drug Review (CDR) and on the opinion of an out-of-province specialist, who questions the effectiveness of the drug in children older than five.

The CDR considers how a new drug compares with alternatives, which patients will benefit and whether it will deliver value for money.

The Akhter children, who are eight, 10 and 12 years old, all began showing symptoms around age four or five, but doctors were unable to diagnose the problem until 2012, when all three were diagnosed in Winnipeg, their father said.

An advocate says the ministry’s decision was based on the drug cost rather than evidence about its effectiveness.

“We are really pushing the minister to make the decision based on the clinical data that shows the earlier treatment begins, the better, but there is no age limitations on which treatment can be beneficial,” said Jaime Myrah, executive director of the Canadian MPS society.

“All of the clinical trials took place in patients above the age of five, so there is actually no clinical data that suggests the treatment is more effective in children under the age of five. We do believe this is really a decision that is being made by financial considerations,” Myrah said.

The disease is estimated to appear in only one out of every 200,000 to 300,000 births.

The Akhter children’s bones and joints are weak, as are their hearts and lungs, and they have all suffered vision and hearing loss. They have also stopped growing.

Akhter wasn’t expecting the refusal to fund, given that the treatment is covered for one other child in Saskatchewan.

“My wife and I, we were very sad,” he said. “We are trying our best to get that medicine, to see them happy, healthy.”

Doctors have told Akhter the children can expect a lifespan of 20 to 30 years – and they’ll become more dependent as the disease continues to take its toll.

Myrah said patients have also been approved in Ontario, Quebec and soon Alberta. Saskatchewan and other provinces have approved treatments similar to Vimizim, she said.

With Betty Ann Adam files

jcharlton@thestarphoenix.com

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/

Minister Refuses To Allow Renowned MPS Expert To Join Family Meeting; Doucet’s Family In Fredericton to Meet with Child And Youth Advocate Thursday

Ministry of Health Reverses Funding Decision; Family Elated That “Hope Has Returned”

Expert says drug would work well for Akhter children

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Approval ‘the hope of our life’

Photograph by: Gord Waldner, The Starphoenix , The Starphoenix