The Isaac Foundation
Request for Collaborative Approach – An Open Letter To Flemming Ornskov
Dear Mr. Ornskov and Mr. Vickers,
Andrew McFadyen
Meeting With Shire – Update
Hi Everyone,
It’s with a heavy heart, and with anger and frustration, that I update you on the meeting that took place this morning between the Fowler family, The Isaac Foundation, and Shire Pharmaceuticals. The meeting was organized after Shire’s decision to deny Jack Fowler the life saving treatment he immediately requires.
This meeting was supposed to take place at the Fowler home, 45 minutes outside of the city of Chicago. It was set to take place at 1:30 p.m. However, a few days before the meeting, Shire’s CEO Flemming Ornskov abruptly changed the location of the meeting to the Chicago airport, and changed the time to an unseemly 7:30 am. The Fowler family struggled to put child care in place. And when you are dealing with a special-needs child, that is no easy task.
We made it to the meeting on time, and brought Jack in with us to meet with the Shire team. Present members for Shire were CEO Flemming Ornskov and Head of Research and Development, Phil Vickers.
The Isaac Foundation began the meeting by thanking Shire for taking the time to meet with the Fowler family. We expressed that the purpose of the meeting from our point of view was to discover how we could work collaboratively with Shire Pharmaceuticals so that we can find the best treatment options for Jack Fowler and in a timely fashion. We expressed that if Shire was present to simply reiterate their position from December and deny Jack the treatment he needs, then the meeting would need to come to an abrupt end. I made very clear to Mr. Ornskov one very simple fact – an immoral decision is still immoral, even if it’s delivered while looking us in the eye and said to our face.
Flemming looked at us and said “We are not changing our decision. I guess this meeting is over.” With that, the hopes of the Fowler family were dashed, and our hope to work together with Shire to save this little boy was ended. We left a large print out of the 32,000 signatures that were signed in the online petition, as well as letters of support from a high percentage of the families currently participating in the clinical trial of the drug that Jack desperately needs.
As we were leaving, Jack walked around the table and gave Flemming a hug. Close to tears, Jack’s mom said “If he could talk, he would be asking you to Be Brave, like your motto says, and save him.” It was one of the most heartbreaking moments of my life – watching a sweet little boy who doesn’t have any idea what is going on give a hug to the man who just gave him a certain death sentence. I told Flemming that I had a hard time understanding how he can go home and look his children in the face after that moment. He just looked at us and said “It was nice to meet the family.”
The facts of this case remain the same, and are very clear. Jack Fowler needs access to a drug that will save his life. He easily qualifies for individual use access through the FDA’s Expanded Use guidelines. It is those guidelines that decide whether any investigational drug is safe for use outside the bounds of a clinical trial. The question of whether enough safety data exists to proceed or not doesn’t rest with Shire Pharmaceuticals, nor does it rest with any pharmaceutical undergoing the same process. It rests with the FDA first and foremost, and it rests with the physician in charge of treating the patient. All Shire has to do is begin the application process on Jack’s behalf and leave the decision to the FDA. When I stated this very clearly to Mr. Ornskov and asked if he would submit the application, he flatly refused.
There are things in this world that many people would be better off not knowing. What lurks in the minds of pharmaceutical decision makers should be at the top of everyone’s list. To have the ability to provide help and support, to be able to save the life of a precious little boy, and then choose not to, shows a callous disregard toward life. That callous disregard is a painful reminder of what Big Pharma is after – money, product, fortune, and fame. Don’t ever be fooled that the patient comes first. As Flemming so plainly stated before I gave my introduction – “We don’t work with patients. We don’t work with families. We are in the business of developing product.”
With that, there’s nothing left to be said.
Our press release goes out early this week. Stay tuned on how you can help us #SaveJack. We will never quit when the life of a child hangs in the balance.
Thank you for your ongoing and tremendous support.
With Love,
The Isaac Foundation
Jack’s Story
Hi Everyone,
Late last week, I received a note from Jamie Fowler, a fellow MPS parent who lives in the US. The email was entitled “Help!” and, needless to say, left me heartbroken. It was a plea for help, for support, a last ditch effort to anyone listening to help save her son, Jack. Jack suffers from MPS II and has been bravely fighting the disease as best he can. Of late, the disease has been relentlessly attacking Jack’s brain, and he has been regressing at a rapid pace.
To any parent, to any decent human being, this is heartbreaking. Please read Jamie’s email below, and then continue reading to see how you can help Jack receive the treatment he so desperately needs.
From Jack’s Mom
Another sleepless night…
Tonight I found Jason on the kitchen floor sobbing. I’m in bed ready to tackle sleep and trying to contain my emotions, then I hear the sound of my solid rock husband Releasing. He had found pictures of our spunky Jack that he hadn’t seen for awhile. Jack getting into the cabinets, cooking and mixing. Jack playing the flute and drums. Jack playing in his fire station set. Jack exploring in the sandbox. Jack dancing and singing show tunes. And the memories go on. It’s incredibly challenging to share and exposure you ladies to what I’m dealing with at my end. You both go to bed knowing your sons have a chance at life, which lightens me to know, but inevitably I end up going to bed crying from watching my son lose another skill set, a step closer to death. I share bits and pieces but the reality is I struggle every moment of every single day with fear. Fear of this final phase of regressing, losing ALL his skills. Boy do I feel alone. Yesterday Jack stared at me with spoon in his hand, no idea what to do with it. His favorite entertainment, the iPad, he is no longer able to operate it. The list goes on and on. I put on a smile for him and Juliet and “pretend” tomorrow will be better only to find tomorrow is always worse.
He stared at me last weekend while sitting in the bath, boy do I miss him being feisty -yelling and splashing me, now he sits and stares as if he wants to do or say something to me but just can’t. It’s way beyond heartbreaking.But back to Jason in the kitchen. After a long hard cry we discussed our sorrow and how we must cope while moving through this horrific phase with no easy solution for help. And then we both reminded each other our promise we made on Diagnosis Day that we would do ANYTHING and EVERYTHING to make Jack happy and comfortable. Jason feels failure and I just don’t want to ever look back on this time with what ifs.
Each day is another day Jack is gone.
We know there is drug. We know it is safe. We know Jack needs it!
With a heavy and light (yes both),
Jamie.
We have an incredible team that is committed to moving forward swiftly on behalf of Jack. We will be doing everything we can to ensure that Jack gets the treatment that he needs. What is that treatment? Currently, Shire is undertaking a clinical trial where they direct the treatment MPS II sufferers need directly into the brain. The results, so far, have been incredibly positive. Unfortunately, Jack doesn’t qualify for the trial because he has high Intracranial Pressure in his brain – Pressure that would be corrected with the treatment. A sadly ironic situation, but one that can be overcome.
Currently, we have a site where Jack can receive the treatment. We have a physician that can administer the treatment, and we’re confident that the Independent Review Board will approve administering it to Jack. The only thing we need is access to the drug on a compassionate basis. Simply put, without that access, Jack will die.
And I won’t sit back and allow that to happen.
We’ll need our team over the coming weeks and months. I hope to work with Shire to develop a plan to collaboratively work together to help Jack. I’ll drop by here and on our social media pages to update things as they progress.
Hang in there with us, Jack. We’re doing everything we can to help.
With Love,
A.
Jack's Story
Hi Everyone,
Late last week, I received a note from Jamie Fowler, a fellow MPS parent who lives in the US. The email was entitled “Help!” and, needless to say, left me heartbroken. It was a plea for help, for support, a last ditch effort to anyone listening to help save her son, Jack. Jack suffers from MPS II and has been bravely fighting the disease as best he can. Of late, the disease has been relentlessly attacking Jack’s brain, and he has been regressing at a rapid pace.
To any parent, to any decent human being, this is heartbreaking. Please read Jamie’s email below, and then continue reading to see how you can help Jack receive the treatment he so desperately needs.
From Jack’s Mom
Another sleepless night…
Tonight I found Jason on the kitchen floor sobbing. I’m in bed ready to tackle sleep and trying to contain my emotions, then I hear the sound of my solid rock husband Releasing. He had found pictures of our spunky Jack that he hadn’t seen for awhile. Jack getting into the cabinets, cooking and mixing. Jack playing the flute and drums. Jack playing in his fire station set. Jack exploring in the sandbox. Jack dancing and singing show tunes. And the memories go on. It’s incredibly challenging to share and exposure you ladies to what I’m dealing with at my end. You both go to bed knowing your sons have a chance at life, which lightens me to know, but inevitably I end up going to bed crying from watching my son lose another skill set, a step closer to death. I share bits and pieces but the reality is I struggle every moment of every single day with fear. Fear of this final phase of regressing, losing ALL his skills. Boy do I feel alone. Yesterday Jack stared at me with spoon in his hand, no idea what to do with it. His favorite entertainment, the iPad, he is no longer able to operate it. The list goes on and on. I put on a smile for him and Juliet and “pretend” tomorrow will be better only to find tomorrow is always worse.
He stared at me last weekend while sitting in the bath, boy do I miss him being feisty -yelling and splashing me, now he sits and stares as if he wants to do or say something to me but just can’t. It’s way beyond heartbreaking.But back to Jason in the kitchen. After a long hard cry we discussed our sorrow and how we must cope while moving through this horrific phase with no easy solution for help. And then we both reminded each other our promise we made on Diagnosis Day that we would do ANYTHING and EVERYTHING to make Jack happy and comfortable. Jason feels failure and I just don’t want to ever look back on this time with what ifs.
Each day is another day Jack is gone.
We know there is drug. We know it is safe. We know Jack needs it!
With a heavy and light (yes both),
Jamie.
We have an incredible team that is committed to moving forward swiftly on behalf of Jack. We will be doing everything we can to ensure that Jack gets the treatment that he needs. What is that treatment? Currently, Shire is undertaking a clinical trial where they direct the treatment MPS II sufferers need directly into the brain. The results, so far, have been incredibly positive. Unfortunately, Jack doesn’t qualify for the trial because he has high Intracranial Pressure in his brain – Pressure that would be corrected with the treatment. A sadly ironic situation, but one that can be overcome.
Currently, we have a site where Jack can receive the treatment. We have a physician that can administer the treatment, and we’re confident that the Independent Review Board will approve administering it to Jack. The only thing we need is access to the drug on a compassionate basis. Simply put, without that access, Jack will die.
And I won’t sit back and allow that to happen.
We’ll need our team over the coming weeks and months. I hope to work with Shire to develop a plan to collaboratively work together to help Jack. I’ll drop by here and on our social media pages to update things as they progress.
Hang in there with us, Jack. We’re doing everything we can to help.
With Love,
A.
The Pass
I just had a touching moment with Isaac, another one of those beautiful moments when I marvel at how incredibly insightful and thoughtful he is.
Everyone who knows Isaac knows that he loves music. John’s his favourite, followed by Danny Michel and Adele. But he loves all music, and listens intently to the lyrics, often wondering about their meaning. He gets emotional, sometimes, at the sad songs, and he rocks out to the fun ones.
It’s easy for him, too, because we always have music on in the house. Or in the car. Or garage, or outside. Music is everywhere in our life, which is probably why we’ve made it a central component of our Gala For A Cure every year.
A few minutes ago, Isaac popped over and asked “Dad, which song was it that made you want to start The Isaac Foundation?” I was surprised – I haven’t told too many people when and how The Isaac Foundation came into being. But I have told those close to me – probably in one of my many emotional or reflective or “softy” moments. And I should remember that Isaac sees and hears EVERYTHING. It should come as no surprise that he’s heard me tell the story before. But he’s kept it inside; pondering, perhaps, how a song could start someone on the journey we’ve found ourselves on.
I smiled and told him I’d put it on for him. We listened, but he wanted to know EXACTLY what about the song made me want to start our charity, and our search for a cure. We skipped back to the start of the song and listened closely together, reading the lyrics on the screen at the same time. I watched him tear up as he read, and I described the moments in the song that woke me up out of my grieving, the message that got me the hell out of bed and on my way to finding a cure
Whenever I put that song on, I’m always transported back to those days after diagnosis – the awful, dark days when I couldn’t get out of bed. The weeks that I couldn’t eat (I lost 40 pounds quickly). The haunting nightmares I had, the dark, dark places my mind took me to. I remember driving through a dark February night, watching the stars, and trying to escape with some music. And I remember this song coming on and knocking some sense into me. I decided on that night that I wasn’t going to lay around and feel sorry for myself. My son, after all, wasn’t going to get better by me sitting around and waiting for the disease to take hold in him. Simply put, that February night changed my life.
Going back in time to that night always brings such mixed emotion, and it felt a bit surreal sitting beside the boy I’ve worked so hard to save and reliving it.
Isaac and I finished listening to the song together and we sat quietly for a long moment. He looked up at me and said “Dad. Would I be dead right now if you didn’t hear that song?” My heart aching, I told him no – I would have woken up sooner or later, and we would have figured this out either way. But I’m glad I heard that song that night. It’s provided me a turning point I can always look back on, and a reminder to always keep going forward, no matter what (or who) gets in the way.
There’s still many nights I want to cry out “Christ, what have you done?” just like in the song. More nights than I know what to do with. But there’s also many more nights when I remember the Oscar Wilde quote about all of us being stuck in the gutter, and only some of us turning to look at the stars (portrayed in my inspiration song, too).
My boy never ceases to amaze me. He is insightful beyond his years. And as we head into our Gala next weekend, and start our journey with our new Non-Profit (announcement soon!), I’m glad he waited until today to ask me about my inspiration for The Isaac Foundation. I needed to slow down and remember WHY we’re doing what we’re doing, and I’m glad I did.
Song and lyrics are below. See many of you next week in person or via our Live Stream from the Gala.
Thanks, as always, for your support.
With Love,
A.
“The Pass”
Waiting for the world’s applause
Rebel without a conscience
Martyr without a causeStatic on your frequency
Electrical storm in your veins
Raging at unreachable glory
Straining at invisible chainsAnd now you’re trembling on a rocky ledge
Staring down into a heartless sea
Can’t face life on a razor’s edge
Nothing’s what you thought it would be
All of us get lost in the darkness
Dreamers learn to steer by the stars
All of us do time in the gutter
Dreamers turn to look at the cars
Turn around and turn around and turn around
Turn around and walk the razor’s edge
Don’t turn your back
And slam the door on me
It’s not as if this barricade
Blocks the only road
It’s not as if you’re all alone
In wanting to explode
Someone set a bad example
Made surrender seem all right
The act of a noble warrior
Who lost the will to fight
And now you’re trembling on a rocky ledge
Staring down into a heartless sea
Done with life on a razor’s edge
Nothing’s what you thought it would be
No hero in your tragedy
No daring in your escape
No salutes for your surrender
Nothing noble in your fate
Christ, what have you done?
J&J – The Evolution of a Lovely Relationship
Hi All,
I’ve promised myself that I would post new blogs more often that I’ve found time for in the past. It’s been another crazy year, for sure, but if I’m confident I can find 5 occasional minutes to blog now and then!
Just wanted to post a quick update while it was first and foremost on my mind. I spoke with Dr. Ray at JnJ this evening and wanted to note how impressed I continue to be with the team at Janssen Pharmaceuticals and, as we’ve always referred to them as, Johnson and Johnson. Or relationship started off, as you may remember, quite rocky. The Isaac Foundation, along with our partner organizations, had long lobbied the company to commit to research of a highly promising drug (Elmiron) that the company makes and markets. After repeated no answers, and constant put-offs, we put our advocacy efforts on behalf of our kids in high gear. After months of hard work and patience, we finally had the opportunity to present our case to some decision makers within the company. From that moment forward, things changed, as did our view on the company.
Once the potential of Elmiron was recognized by JnJ, the company acted quickly to repair any damage that was done with our organization but, more importantly, with the MPS community as a whole. They committed to further study of their product, brought some of the most incredible MPS researchers and physicians together for a collaborative planning and educational session, and took a keen interest in our children. They put a team together that dedicated themselves to learning about MPS and the struggles families dealing with this devastating disease face, something that I was incredibly impressed with, and continue to be proud of.
What impressed me most was that they didn’t take a serious look at this drug for financial reasons – JnJ stood to lose a lot of money during the entire endeavour. They chose to look into this product because the data was compelling, and the impact it could have on saving our children was undeniable. We often took aim at the JnJ Credo Values – values the company promoted and prided themselves on. Once Dr. Amrit Ray took over the Elmiron/MPS file, that credo was put into action and the company showed me why they put such stock in their credo values. In short – they undertook this project because it was going to positively impact the lives of our kids, and they felt it was the right thing to do.
Unfortunately, JnJ was prevented from further study of Elmiron due to legal issues relating to patents, along with Mt. Sinai’s deal with another drug company. However, I’ve been fortunate to continue working with Dr. Ray and have had the opportunity to touch base with him on a regular basis. He and his team continue to learn about MPS and our children, and I’m incredibly touched at how caring and compassionate he and his team have been.
I spoke with Dr. Ray this evening. And like every time we chat, I was struck by how empathetic he is as it relates to our kids. Johnson and Johnson is lucky to have him at the helm, and we at The Isaac Foundation are lucky that our relationship has evolved over time to find us at the place we are today.
Thanks, as always, for the support you provide us.
With Love,
A.
J&J – The Evolution of a Lovely Relationship
Hi All,
I’ve promised myself that I would post new blogs more often that I’ve found time for in the past. It’s been another crazy year, for sure, but if I’m confident I can find 5 occasional minutes to blog now and then!
Just wanted to post a quick update while it was first and foremost on my mind. I spoke with Dr. Ray at JnJ this evening and wanted to note how impressed I continue to be with the team at Janssen Pharmaceuticals and, as we’ve always referred to them as, Johnson and Johnson. Or relationship started off, as you may remember, quite rocky. The Isaac Foundation, along with our partner organizations, had long lobbied the company to commit to research of a highly promising drug (Elmiron) that the company makes and markets. After repeated no answers, and constant put-offs, we put our advocacy efforts on behalf of our kids in high gear. After months of hard work and patience, we finally had the opportunity to present our case to some decision makers within the company. From that moment forward, things changed, as did our view on the company.
Once the potential of Elmiron was recognized by JnJ, the company acted quickly to repair any damage that was done with our organization but, more importantly, with the MPS community as a whole. They committed to further study of their product, brought some of the most incredible MPS researchers and physicians together for a collaborative planning and educational session, and took a keen interest in our children. They put a team together that dedicated themselves to learning about MPS and the struggles families dealing with this devastating disease face, something that I was incredibly impressed with, and continue to be proud of.
What impressed me most was that they didn’t take a serious look at this drug for financial reasons – JnJ stood to lose a lot of money during the entire endeavour. They chose to look into this product because the data was compelling, and the impact it could have on saving our children was undeniable. We often took aim at the JnJ Credo Values – values the company promoted and prided themselves on. Once Dr. Amrit Ray took over the Elmiron/MPS file, that credo was put into action and the company showed me why they put such stock in their credo values. In short – they undertook this project because it was going to positively impact the lives of our kids, and they felt it was the right thing to do.
Unfortunately, JnJ was prevented from further study of Elmiron due to legal issues relating to patents, along with Mt. Sinai’s deal with another drug company. However, I’ve been fortunate to continue working with Dr. Ray and have had the opportunity to touch base with him on a regular basis. He and his team continue to learn about MPS and our children, and I’m incredibly touched at how caring and compassionate he and his team have been.
I spoke with Dr. Ray this evening. And like every time we chat, I was struck by how empathetic he is as it relates to our kids. Johnson and Johnson is lucky to have him at the helm, and we at The Isaac Foundation are lucky that our relationship has evolved over time to find us at the place we are today.
Thanks, as always, for the support you provide us.
With Love,
A.
Thank You RBC and Arthur Lions Club!
The Isaac Foundation extends a huge thank you to the Arthur Lions Club and RBC for their incredibly generous donation to The Isaac Foundation through our satellite office with the More Family. All funds donated will go directly to funding research and finding a cure for this rare and devastating disease.
The photo to the left shows Darren More with Jasper, accepting the generous donation from the Club.
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.”
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.