October 5, 2015
Source: http://saskatoon.ctvnews.ca/video?clipId=720671
Advocacy
Morquio A Syndrome (MPS IVA) Advocacy
Hi Everyone,
I’m excited to announce that The Isaac Foundation will be working with families throughout Canada to help ensure provincial funding for Enzyme Replacement Therapy (ERT) for MPS IVA patients is forthcoming once the treatment gets approval from Health Canada.
Morquio Syndrome is a rare disease that is part of the MPS umbrella of lysosomal storage disorders. The disease affects major organ systems in the body and leads to devastating symptoms for sufferers. These symptoms included bone and joint disease, heart and airway disease, a shortened stature, and premature death.
We have been fortunate to have had success advocating for families seeking support and treatment options for other forms of MPS in numerous provinces over the past 8 years. Currently, there are no treatment options for sufferers of MPS IVA. However, recent clinical trials for Biomarin’s Vimizim have produced very promising results, and Health Canada is expected to approve the treatment for use in Canada in the very near future – most likely before the summer of 2014. A decision by the FDA in the United States is imminent, and an approval in the US could mean that patients can access the treatment via the Federal Government’s Special Access Program. However, approval through the SAP is always contingent on provincial funding being made available.
We are excited to help advocate with families as they seek treatment options for themselves or their affected children, and will work with the same passion and resolve that we have given all of our other advocacy efforts.
If you or your family is interested in working with The Isaac Foundation to help bring ERT to your province for yourself or your children, please don’t hesitate to contact Andrew McFadyen at HERE or via telephone at 613.328.9136.
For more information about Morquio A Syndrome please click HERE.
Boston Globe – A Few Notes
Hi Everyone,
Jack Fowler’s case was presented in the Boston Globe today. And while it’s nice to get some exposure, I truly feel the editor at the Globe should be held accountable for the biased and single-sided view the article portrayed.
Nowhere in the article was the FDA’s position put forward – a position we have been open and transparent about presenting since day 1. That position is simple – access to investigational medicines outside of the clinical trial setting have NO BEARING on clinical trial results. In fact, there are cases where expanded use have helped to speed up the process. Indeed, the FDA guidelines say the following:
“There are examples in which FDA has made use of adverse events information from expanded access use in the safety assessment of a drug. There are a small number of cases in which an important adverse event was first identified during expanded access use and those adverse events were in clouded in product labelling. This is not a negative from a public health perspective – the sooner important adverse events are identified the better. Even from the sponsor’s viewpoint, early discovery of a rare adverse event is, on the whole, a benefit. Although adverse events first identified during expanded use of a certain drugs have been included in the drugs’ approval product labelling, we are unaware of any cases in which adverse event information obtained from expanded access use has resulted in denial of approval for a product.”
As I have previously noted, the FDA has been in contact with The Isaac Foundation regarding expanded access, and I have assurances that any adverse events that happen outside of the clinical trial setting will not hinder the clinical trial for any reason. Why the Boston Globe editor did not see fit to ensure that information was presented is beyond me, is unfortunate, and leaves an incomplete picture of the story as a whole.
In addition, no quotes were sought from families who have children currently participating in the trial. The trial is currently in Phase II/III and has lasted over 4 years. Families are reporting incredible results in their children – their lives have been saved. The treatment works, and the reversal of devastating symptoms these children have been experience is proof positive. Moreover, the treatment is safe! Over 4 years of clinical trial has seen NO ADVERSE EVENTS REPORTED. None. Zero. A few words from families who have experience dealing with this treatment would have shed light on the trial, the effectiveness of the treatment, and the safety of administering that treatment to our kids.
Finally, with all due respect to Dr. Muenzer, I have to object to his comment that “compassionate use will destroy a trial.” That statement flies in the face of all evidence the FDA has provided us, and flies in the face of the entire purpose of compassionate use for our kids. Expanded use guidelines were clarified by the FDA a few short years ago, and they were clarified so that physicians and pharmaceutical companies alike could fully understand that compassionate use does not affect trials. Their guidelines ensure that expanded access is provided as a treatment option for patients, not a data collection option for principal investigators or pharmaceutical companies. In short – any expanded use will not impact the trial. Not by any stretch. And to further this point, I emailed Janet Woodcock at the FDA for a direct statement on the matter. She promptly replied and said “…in our collective knowledge here at CDER, adverse events occurring during the development program have not delayed the programs. In one case, we know the drug development was actually accelerated.” One would have expected this information would be available to anyone who asked, including Shire Pharmaceuticals.
It is beyond me why the Boston Globe would neglect to present these viewpoints in their story. Journalism is supposed to present an objective viewpoint on a trending topic. It’s purview is to educate the public and allow for open discussion. For that discussion to take place – for any fair and open discussion to take place – all the viewpoints need to be laid out. Sadly, important information was precluded from the story, and I hope this entry helps clarify the matter once again.
Finally – Phil Vickers – never in this world would it be “ethically wrong” to try to save a child’s life. EVER.
On another note, I emailed Shire CEO Flemming Ornskov yesterday to ask, once again, for a collaborative solution to this matter. I am hopeful that Flemming and his team will agree to sit down directly with the FDA, Dr. Muenzer, and Dr. Burton to work through this matter, set a proper policy and guidelines for access to this investigational drug, and work to do what is best for our children. ALL of our children.
With thanks for your continued support,
The Isaac Foundation
Request for Information – Shire plc
Hi Everyone,
Below is a note I sent to Shire CEO Flemming Ornskov, requesting information we’ve been waiting on for the past week. Outlined is our rationale why Jack should have access to expanded use of IT ERT. After being very open with communication while they were deciding Jack’s fate, Shire has decided not to provide us with any requested information after they decided to deny Jack a fair chance at life.
I am posting this here because we have been inundated with requests for updates from families throughout the US and Canada. I will do my best to ensure all information is made readily available. I appreciate the time everyone has taken to contact us and the help and support you’ve offered.
We are speaking with the Shire team tomorrow evening. I’ll post any news as it becomes available. In the meantime, send Jack your words of Hope, and send Shire a message telling them you support Treatment For Jack HERE.
Saturday, December 14, 2013
Dear Mr. Ornskov,
On Monday of last week, I requested the criteria that Shire uses to determine which candidates, if any, qualify for pre-approval access to an investigational drug. I have made two other requests for the information in the subsequent days. As of yet, we have not received that criteria. It would be helpful for my organization, as well as Dr. Barbara Burton, to be able to review those criteria before we speak with your team on Monday, December 16th. I am writing, once again, to request that those criteria be made available. Successful companies that develop therapies which can potentially and dramatically enhance the quality of life for individuals traditionally have pre-set conditions with which patients qualify for EAP use and I am confident that Jack meets the criteria your company has created.
In addition, I firmly believe that Jack meets the criteria set forth by the FDA for approval for any expanded use of your therapy. As I have previously stated, I understand your concern about the number of children you currently have safety data on (15). In the rare disease world, this is a high number. With the promising results thus far, it is our opinion that this treatment would easily qualify under the FDA’s Breakthrough Therapy designation and could be fast tracked in short order. Providing access to Jack has the potential to bolster that case even further and should be considered so that all sufferers of Hunter Syndrome can be treated sooner.
The facts of this case are clear: we have a child who is in desperate need of treatment. We have an available treatment that has the potential to save his life. That child qualifies under the FDA’s criteria set forth for pre-approval access to an investigational drug. We have a physician who is anxious to treat Jack as soon as possible, and we have an independent review board that we are confident will approve such treatment.
It is clear that this decision comes down to a question of values. Specifically, will Shire live up to its stated objectives of “keeping the patient at the heart of everything we do”? Will you choose to “Be Brave”, as your responsibility creedo dictates you aim to be for your patients? Or will you abandon your responsibility to help those you can when they are most in need?
The MPS community is shocked, angered, and disheartened by the news released last week regarding treatment for Jack. Many participants currently enrolled in your clinical trial are organizing a boycott because they feel abandoned and controlled by a pharmaceutical company clearly protecting their own best interests. I’ve been contacted by numerous families throughout Canada and the United States questioning whether you believe in your study – whether you believe in the treatment you have under development. Denial of access to treatment for Jack shows many that you don’t, in fact, believe in its potential. If you did, action would be taken immediately to save the life of a child that only you can save.
Please pass along your EAP criteria as soon as possible so that we may review it prior to Monday’s meeting. If you would like to speak in person regarding that criteria or to update me on when I can expect it, please don’t hesitate to contact me anytime at 613.328.9136. If no such criteria exists within Shire, we are happy to work with you to help bring such a policy to fruition. Creating life-altering treatments comes with a responsibility to make it available to those most in need, and we would welcome an opportunity to work collaboratively to ensure those needs are met.
Sincerely,
Andrew McFadyen
The Isaac Foundation
“Love, Laughter, and Hope”
www.theisaacfoundation.com
Alberta will help fund critical treatment for young St. Albert girl
BY SARAH O’DONNELL, EDMONTON JOURNAL AUGUST 13, 2013 7:34 AM
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.”
© Copyright (c) The Edmonton Journal
Aleena Sadownyk will have treatment covered by Alberta government
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.
Aleena Sadownyk Treatment: Alberta To Pay For Drugs For 3-Year-Old With Rare Enzyme Deficiency
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.
ERT – Proof of Effectiveness From Around The World
The question of whether Alberta Health will fund treatment for Aleena will be based solely on whether the province wants to pay the high cost associated with their decision. How do I know this? Well – if they were basing their decision on evidence based science alone, they would have rendered their decision by now – and that decision would have to be a YES. To support that claim, I thought I’d take the time to include some snippets from some of the finest MPS Researchers in the world. These quotes are taken directly from readily-available and reputable Journals, with sources included for further study. There is irrefutable proof that this treatment is effective. There is irrefutable proof that this treatment MUST begin at a very early stage. Delaying a decision on providing this treatment for Aleena is detrimental to her long-term health!
Here’s some things to ponder and share. It took me less than 1/2 day to do this review of the available scientific literature regarding the safety and effectiveness that ERT yields patients suffering from MPS VI. How it’s taken Alberta Health over 4 weeks to find this information is beyond comprehension. And if they didn’t find this information during their STEDT review, they haven’t done a thorough job evaluating this case at all! I should note that this is just a small sampling of the literature that is available on the subject, and every article I dug up promotes the same overall message – ERT is the best treatment for children suffering from MPS VI, it’s EFFECTIVE, and it must begin EARLY. One final note – all articles point to this treatment being SAFE. With all this clinical data available, Alberta Health must simply be weighing whether they want to pay to save Aleena or not, and shame on them if that’s the case.
Please read and share. #Treatment4Aleena
“Recently, a consensus panel of international experts in medicine, genetics and biochemistry drafted management guidelines for MPS VI. The expert panel recommended ERT, when available, as first-line therapy.” (Schlander, M. and Beck, M. Current Medical Research and Opinion, 2009)
“Within 24 weeks of treatment, most patients treated with ERT demonstrated significant and sustained improvements in performance in [6 and 12 minute walking tests]. Long-term safety data show that the therapy has an acceptable safety profile.” (Harmatz, P. Turkish Journal of Pediatrics, 2010)
“…initiating ERT at an early age is safe and improves overall morphology, clinical outcome, quality of life and the safety profile related to immune response. The main benefit was in scoliosis, joint range of movement, cardiac valves and facial appearance.” (McGill JJ, Inwood AC, Coman DJ, Lipke ML, de Lore D, Swiedler SJ, Hopwood JJ. Clinical Genetics, 2010)
“As ERT slows down the accumulation of GAG in cells and tissues, it is thought that early treatment might prevent or delay the development of irreversible disease manifestations and limit or prevent growth deceleration.” (Harmatz, P. Turkish Journal of Pediatrics, 2010)
“…this trend toward decline in pulmonary function can be halted and partially reversed during ERT…” (Harmatz, P., Yo, Z., Giugliana, R., Schwartz, V., Guffon, N., Teles, E., Miranda, C…Decker, C. Journal of Inherited Metabolic Disease, 2009)
“One recently published case control study assessed the impact of [ERT] in two siblings: one treated from the age of 8 weeks, one from 3.6 years. After 3.6 years of treatment with [ERT], the youngest child had a lack of scoliosis and preserved joint movement, cardiac valves and facial morphology, unlike the older sibling at the same age. The older sibling had improvements in joint mobility and cardiac valve disease after 3.6 years of treatment with [ERT].” (Harmatz, P. Turkish Journal of Pediatrics, 2010)
“…it seems desirable to start treating these patients at an early stage, before irreversible damage has occurred.” (Schlander, M. and Beck, M. Current Medical Research and Opinion, 2009)
“[ERT] was found to be safe and effective in this young patient population and similar to that seen in clinical trials with older patients.” (Horovitz, D., Magalhaes, T., Acosta, A., Ribeiro, E., Giuliani, L., Palhares, D., Chong, K…Llerena Jr., J. Molecular Genetics and Metabolism, 2013)
“This improvement in respiratory function relative to baseline may lead to a decrease in the severity of respiratory illnesses and number of hospitalizations, and an overal improvement in the quality of life of MPS VI patients.” (Harmatz, P., Yo, Z., Giugliana, R., Schwartz, V., Guffon, N., Teles, E., Miranda, C…Decker, C. Journal of Inherited Metabolic Disease, 2009)
“This therapy opens the door to a more proactive approach of managing the disease, i.e slowing down the accululation of GAG rather than alleviating the resulting clinical manifestations.” (Harmatz, P. Turkish Journal of Pediatrics, 2010)
“…conventional cost-effectiveness criterion currently in widespread use does not offer sufficient basis for rejecting reimbursement of expensive treatments for exceptionally rare disorders.” (Schlander, M. and Beck, M. Current Medical Research and Opinion, 2009)
“The prescribed dosage of 1 mg/kg IV weekly with galsulfase ERT is shown to be safe and effective in slowing and/or improving certain aspects of the disease.” (Horovitz, D., Magalhaes, T., Acosta, A., Ribeiro, E., Giuliani, L., Palhares, D., Chong, K…Llerena Jr., J. Molecular Genetics and Metabolism, 2013)
“[ERT] weekly has shown to be safe and effective in slowing progression and/or improving the burden of the disease for MPS VI in young children. As early treatment initiation results in improved patient outcomes in this young cohort, early recognition of the more subtle symptoms associated with slowly progressing disease should be a priority to ensure early diagnosis and treatment initiation.” (Horovitz, D., Magalhaes, T., Acosta, A., Ribeiro, E., Giuliani, L., Palhares, D., Chong, K…Llerena Jr., J. Molecular Genetics and Metabolism, 2013)
“ERT positively affected mobility of the shoulder joint, the size of the liver and spleen, cardiac parameters, pulmonary function, certain domains of [quality of life], and the level of GAGs in the urine.” (Brands, M. Oussoren, E., Ruijter, G., Vollebregt, A., van den Hout, H., Joosten, K., Hop, W., Plug, I., Ploeg, A. Molecular Genetics and Metabolism, 2013)
“…results of ERT treatment in MPS VI have been promising, demonstrating clinically and statistically significant improvements in endurance along with a reduction in urinary GAGs.” (Harmatz, P., Giugliani, R., Schwartz, I., Guffon, N., Teles, E., Miranda, M., Wraith, J…Decker, C. Molecular Genetics and Metabolism, 2008)
Editorial: Life-saving treatment a worthwhile investment
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
PROVINCE MUST FUND TREATMENT TO SAVE YOUNG GIRL IN ST. ALBERT: NDP
FOR IMMEDIATE RELEASE
AUGUST 7, 2013
PROVINCE MUST FUND TREATMENT TO SAVE YOUNG GIRL IN ST. ALBERT: NDP
EDMONTON – Today, New Democrat health critic David Eggen sent a letter to Minister Fred Horne requesting that he immediately approve funding for Aleena Sadownyk, a St. Albert toddler, who requires life-saving medical treatment. Eggen is also calling on the PC government to list the necessary treatment for future patients.
“This is another sad and stark example of the PCs’ approach to health care—they prefer to diminish and delist services instead of strengthening and expanding them,” said Eggen. “As a result, this young girl and her family have been forced into a crisis constructed from PC neglect.”
Aleena suffers from the rare enzyme deficiency disease MPS VI, and the treatment, Naglazyme, is not currently funded by the province. Previous attempts by the family to secure funding for Aleena’s treatment have been unsuccessful.
“The PCs must immediately fund treatment for this young girl,” said Eggen. “Beyond that, the Minister needs to commit to funding treatment for others in the future.”
A copy of Eggen’s letter to the Minister is attached. (HERE)
For more information, please contact:
Brad Lafortune, Communications Officer: 780-446-2375 or Bradley.Lafortune@assembly.ab.ca<mailto:Bradley.Lafortune@assembly.ab.ca>
PROVINCE MUST FUND TREATMENT TO SAVE YOUNG GIRL IN ST. ALBERT: NDP
FOR IMMEDIATE RELEASE
AUGUST 7, 2013
PROVINCE MUST FUND TREATMENT TO SAVE YOUNG GIRL IN ST. ALBERT: NDP
EDMONTON – Today, New Democrat health critic David Eggen sent a letter to Minister Fred Horne requesting that he immediately approve funding for Aleena Sadownyk, a St. Albert toddler, who requires life-saving medical treatment. Eggen is also calling on the PC government to list the necessary treatment for future patients.
“This is another sad and stark example of the PCs’ approach to health care—they prefer to diminish and delist services instead of strengthening and expanding them,” said Eggen. “As a result, this young girl and her family have been forced into a crisis constructed from PC neglect.”
Aleena suffers from the rare enzyme deficiency disease MPS VI, and the treatment, Naglazyme, is not currently funded by the province. Previous attempts by the family to secure funding for Aleena’s treatment have been unsuccessful.
“The PCs must immediately fund treatment for this young girl,” said Eggen. “Beyond that, the Minister needs to commit to funding treatment for others in the future.”
A copy of Eggen’s letter to the Minister is attached. (HERE)
For more information, please contact:
Brad Lafortune, Communications Officer: 780-446-2375 or Bradley.Lafortune@assembly.ab.ca<mailto:Bradley.Lafortune@assembly.ab.ca>