I’ve just returned from one of the most rewarding weeks I’ve ever had in my role as Executive Director of The Isaac Foundation. I’ll save updating on all that was the MPS and Adulthood Conference for another blog posting. For now, I wanted to give you an update on Isaac’s health as the conference, for me, was overshadowed by the news we received yesterday (and while I was still away) that Isaac requires and will be undergoing double corneal transplants soon.
This news was shocking and upsetting to us, but not surprising. We’ve always known that this was on the horizon – or that the possibility of this was on the horizon. But he’s been so stable in his health during the past 6 months that it left our minds as a possibility. But stable isn’t quite the right word – he’s improved since January, improved considerably. I guess this is why the news we got yesterday was a tough to take.
First off – Isaac’s being incredibly brave. Please know that. Me? Not so much, but I’ll stay strong because it’s what I have to do. He expressed very eloquently that how his eyes are important to him because he need to be able to read (he’s a veracious reader and it’s unimaginable to him that that could be put in jeopardy.) He wants the surgery to protect that hobby, which is both incredibly cute and heartbreaking.
Now – corneal transplant…what is this and why are we here now?
Kids with MPS accumulate a buildup of cellular waste in their bodies known as glycosaminoglycans, or GAGS. Enzyme Replacement Therapy (ERT) helps to break those GAGS up and clear them out of the body. But sometimes ERT doesn’t prevent the clouding (caused by the GAGS) that takes place in the eyes. And Isaac has always had corneal clouding. Quite severe, in fact, but it’s been severe since he was very young. In fact, if you were to look at Isaac’s eyes today, they don’t have much colour – they are grey due to the clouding.
Isaac’s glasses have corrected his vision over the course of the past numbers of years. The clouding has continued to get worse – to the point where the doctors haven’t been able to see into his eyes for about 2 years. But still – his vision has been stable. A big change happened, however, over the past 6 months. Vision in his left eye has deteriorated considerably – 20/30 down to 20/80, and this is quite concerning. The result? A decision to move on with the transplants that we thought were still a number of years off, in the very least.
Doctors are very good at doing this surgery, though I have to admit that I’m still very scared and worried. But our MPS community is amazing, and many parents are rallying around us with love and support – something I’m truly grateful for. And Isaac’s friend since childhood, who also suffers from MPS, has had both of her cornea transplanted. Her family will be a great resource for us as we embark on this latest battle, and she will be able to talk to Isaac about what to expect.
And quite serendipitous – when I arrived home, there was an invitation in my email for me to tour one of the best (if not THE Best) transplant clinic for kids with MPS in the US. I’m excited to attend and, perhaps, garner a bit of information on the process along the way. The clinic specializes in bone-marrow transplants and stem-cell transplants, but they be a wealth of information for me for this comparatively smaller and less serious procedure.
Anyhow – I feel a tad better now that I’ve written this and the decision has been made. It was hard to get the news while I was away. All I wanted to do was hug my boy, struggle together with my family, and be here for each other when we needed it most. It was a tough night last night, and a long flight home. But as soon as I arrived at the school to pick up my boys, they both jumped into my arms and hugged me for what seemed like an eternity. And Isaac looks good, and strong, and – as he always does – brave. He can do this, and so can I.
Isaac shared the news with his best friend at school, Amy. I’m so thankful that he has someone he can trust to talk to, and I’m sure it made him feel better to share the news with his friends.
I’m listening to Danny Michel as I type this blog update – poignant because he’s playing at our upcoming Gala For A Cure. The song below is called “Just The Way I Am”, and it’s providing the perfect soundtrack for my frame of mind right now as I think about Isaac, his bravery, and the joy I know he takes in having a supportive friend to talk to when things get tough. It’s below for you to listen to as well – it really is a beautiful (and perfect) song for this posting.
Thanks for letting me ramble on. I’ll update with more information when I can. Thanks for always being here for us and our kids.
With Love,
A.
Isaac Update – Corneal Transplant October 7th, 2014mcfadyena
We are thrilled to announce that The Isaac Foundation has granted $200,000 in research grants to 3 innovative projects aimed at finding a cure for MPS!
We are proud to award $100,000 to Dr. Alberto Auricchio in Italy for his incredibly exciting work on Gene Therapy. We hope to see this work head to clinical trials in early 2015!
We are also very excited to award 2 $50,000 research grants from the MPS II FUND, under the leadership of Deb Purcell! The recipients of these awards are granted to Dr. Scott McIvor at the University of Minnesota for his work entitled “AAV Mediated IDS Gene Transfer for MPS II” and to Dr. Brian Bigger at the University of Manchester for his work “Evaluating Stem Cell Gene Therapy for Treating the Brain in MPS II”
This brings the total awarded over the past 6 months to $250,000 – all in hopes of finding a cure for our children.We look forward to seeing results from this research and will continue our work raising money to support these projects and more during the course of the next few years.
Thank you for your incredible support. None of this can happen without you.
2014 Research Grant Award Recipients June 14th, 2014mcfadyena
The Isaac Foundation is proud to announce that our application for our 2014 Research Grants is now available. $100,000 is available from our general MPS Research Fund, and another $50,000 grant is available from the MPS II Research Fund.
Application deadline is May 1. A decision will be reached by June 15, with funds dispersed on or before June 31, 2014.
Should a dying child receive an unproven experimental drug even if the patient doesn’t fit within a carefully designed clinical trial?
More than 53,000 people who have signed an online petition on behalf of a Mundelein boy say yes, absolutely. But Shire, the Ireland-based pharmaceutical company that owns the drug, says it’s a complicated matter and has refused to make the drug available to the child.
At the heart of this moral and ethical dilemma is 6-year-old Jack Fowler. Jack has a rare disorder called Hunter syndrome, or MPS II, that is expected to kill him because he lacks the enzyme needed to break down cellular waste in his body. Ten to 20 years is the usual life expectancy of someone with the disease, but Jack’s more severe type means he may have less time.
An estimated 2,000 patients — nearly all boys — are affected by the disease worldwide.
The hope is that the drug in question, SHP-609, can for the first time, slow or halt the progression of the disease in the brain. It is going through clinical trials, which means it hasn’t been approved by the Food and Drug Administration yet. The Phase II/III stages are just starting, and FDA approval happens after Phase III.
So Jack’s parents, Jason and Jamie Fowler, have been trying to get a “compassionate use” approval for Jack, which the FDA allows on a case-by-case basis. Clearance for such use means Jack’s case would not influence outcomes in the clinical trial, so an adverse reaction by Jack, for example, would not count against the drug.
For two years, the Fowlers have pleaded with Shire to let their son be part of the clinical trial or get the drug through compassionate use, including during a brief meeting with the company’s CEO, Dr. Flemming Ornskov, in a Chicago airport hotel in January. An advocate for the Fowlers, Andrew McFadyen of The Isaac Foundation, a nonprofit group focused on treatments for rare diseases, started the meeting by stating, “An immoral decision remains immoral if delivered in ones face,” and said that if Shire was still choosing death over saving a life then the meeting was over, according to Jamie Fowler.
Shire “delivered the same grim news, so we upped and walked out,” Jamie Fowler said in an email.
Dr. Barbara K. Burton, Jack’s geneticist at Lurie Children’s Hospital of Chicago and one of the investigators for Shire’s clinical trial, said there’s no evidence one way or another that the drug will save Jack’s life. “It’s conceivable that it would hasten his death,” Burton said, but added, “What I do know is that without [any treatment beyond what he’s getting now], he is definitely going to die.”
For that reason, she supports the Fowlers’ position.
Shire said it has compassion for the Fowler family. Yet “we believe that expanding access to SHP-609 beyond the clinical trial can put the overall development at risk and delay or eliminate the opportunity to make a safe, approved treatment more widely available to the global Hunter syndrome community,” Ornskov said.
An FDA spokeswoman said that of the 940 submitted requests for expanded access for such investigational drugs between October 2011 and September 2012, all but four cases were allowed to proceed.
Unknown adverse events usually do not prevent a compassionate use request from being granted, but that is determined on each case, the FDA added. The agency could not comment specifically on Jack’s case.
Dr. Michael Caplan, pediatrics department chairman at NorthShore University HealthSystem, who is knowledgeable about clinical trials, also did not speak about Jack’s situation, but he said experimental drugs usually aren’t given to patients outside clinical trials until Phase III is complete. Safety is the issue, Caplan said.
Shire didn’t say for sure whether that might be a possibility for Jack after the Phase III is done, but a spokeswoman noted that Shire has done that in the past.
Lewis Smith, an associate vice president of research and a medicine professor at Northwestern University Feinberg School of Medicine, said, “these are moral, ethical dilemmas.”
Companies typically are very concerned about allowing people who are not in the clinical trial access to the drug, because “they’re very concerned about whether that would muddy their results, because this is a rare disease,” Smith said.
Yet, it’s an emotional issue, especially when a child is involved, Smith said.
That’s clear when Jamie Fowler talks about how Jack’s cognitive deterioration, which came with the disease, is slowly taking away his personality. Gone is his ability to say “sis” for his sister, Juliet. Fowler dreads the same fate for “mom.” “It’s heartbreaking,” Fowler said, crying.
They’ve turned to social media, such as Facebook, to try to get Shire to reconsider. An online petition hopes to hit 75,000 signatures by the end of the month; there are more than 50,000 names so far. The family also started doing media interviews to get the word out about their case.
Nothing has worked so far, but the Fowlers said they aren’t stopping until Jack gets the drug to see if it can save his life.
“We won’t sit by and watch our son die,” Jamie said.
Contributing: Chris Fusco
Email: mjthomas@suntimes.com
Twitter: @MonifaThomas1
Family fights to get drug for dying 6-year-old son February 20th, 2014mcfadyena
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.
Morquio A Syndrome (MPS IVA) Advocacy February 9th, 2014mcfadyena
Last night on NBC news, Shire Pharmaceuticals released a statement for the media (no personal interviews, sadly). Our response to their statement is included below.
Once again, I’m calling on Shire to publicly address our case, point by point, with their rationale for not supporting expanded access use for Jack Fowler. I’m happy to present the document publicly, and I call on Flemming Ornskov to be open, transparent, and honest with the Fowler family and the public, and provide the reasons that Shire cannot live up to their public motto by “Being Brave” to #SaveJack.
Shire’s statement reads:
We remain deeply sympathetic to Jack Fowler and his family. Patients have been, and will always be, at the heart of what we do, and have driven our desire to research potential new therapies for more than 20 years. Guidance from regulatory agencies is that expanded access to investigational medicines and devices outside of a clinical study setting should be based on the existence of compelling evidence of efficacy, or the drug’s benefit, and safety. Efficacy is typically studied in later stage trials; these data are not yet available for our investigational treatment as we have only just initiated our Phase II/III study. Shire does support, and has provided, expanded access at the appropriate stage in the development process-when clinically validated efficacy and safety information is available. We remain focused on completing our Phase II/III trial with the hope of making our investigational treatment available to as many Hunter syndrome patients as possible, as quickly as possible.
1. “Patients have been, and will always be, at the heart of what we do…”
Flemming Ornskov specifically said to the Fowler family, and I was present to witness this statement: “We don’t deal with patients. We don’t deal with families. We are in the business of developing products.”
2. ” Guidance from regulatory agencies is that expanded access to investigational medicines and devices outside of a clinical study setting should be based on the existence of compelling evidence of efficacy…”
Richard Klein, a member of the Office of Special Health Issues at the FDA (the regulatory body authority that guides expanded access protocol) has stated a few things regarding efficacy (source information at bottom):
“Confidence of safety is more important than efficacy.”
With regards to safety confidence, “for a patient with an immediate life-threatening condition, evidentiary burden is low.”
“Efficacy (and safety) of early phase investigational drugs not proven – and sometimes not known; however, might be given in hope of direct benefit to patient.”
3. “Shire does support, and has provided, expanded access at the appropriate stage in the development process-when clinically validated efficacy and safety information is available.”
Shire did not have a compassionate (or expanded use) policy in place when we approached them about Jack Fowler. I have the transcript of our teleconference where we questioned them on this fact, and they agreed that there should be something in place but that there was not. While they may have allowed this type of request in the past, they have had not set criteria to allow expanded access for patients until January of 2014, long after we approached them to #SaveJack. Incidentally, those criteria are riddled with errors, and I will post our response to that policy soon.
4. “We remain focused on completing our Phase II/III trial…”
Expanded Access to an investigation drug outside the clinical trial setting can HELP with clinical trials and, in some cases, speed up approval of a drug. As well, any adverse affects that transpire while a patient is receiving Expanded Access to a medication DOES NOT IMPACT THE CLINICAL TRIAL in the slightest. See below, direct from the FDA:
In an email to The Isaac Foundation, Janet Woodcock from the FDA stated, “…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.”
The FDA has made a very clear statement on this matter, and included this information in their report on Expanded Access. This report states: “Although adverse events first identified during expanded access use of certain drugs have been included in the drugs’ approved product labeling, 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.”
Former FDA Director Dr. Lester Crawford stated “The FDA, categorically, does not attach special significance to adverse events reported from such expanded access program as (one critic) has tried to join. We recognize that these programs involve less-controlled use of new drugs, and we assess the reported data accordingly. The development of a new medication is not slowed by side effects occurring outside clinical trials.”
Source from Richard Klein: http://www.fda.gov/downloads/ForIndustry/DevelopingProductsforRareDiseasesConditions/OOPDNewsArchive/UCM294794.pdf
Shire has released there guidelines for compassionate use (or expanded use) of their products. They didn’t have any criteria before we approached them, which continues to astound me. Regardless, their criteria is riddled with issues and inaccuracies, and I’ll post our response to it tomorrow.
In the meantime, the info graphic included below is a quote from Shire’s CEO Flemming Ornskov. Jamie Fowler, Jack’s mom, posted it on her page today, and it’s quite telling.
The letter below is posted on Deb Purcell’s blog site over at treypurcell.com. Deb is the director of our MPS II Fund, is a passionate advocate for children suffering from MPS, has been responsible for bringing so much awareness to the disease throughout Canada, and is one of the kindest and most caring people I have ever met.
Deb’s son Trey has MPS II and is currently enrolled in Shire’s clinical trial. Her letter below was hand-delivered to Flemming Ornskov by myself and the Fowler Family during our now infamous 30 second meeting with the CEO.
Sadly, I have a hard time believing that he read it. And I have a hard time believing he took the huge binders with over 32,000 signatures and comments from people around the globe supporting Jack. Or the other letters from parents who have children currently enrolled in the trial. And most sad of all, I expect he didn’t look or read the note that Jack’s mom left him.
Regardless, the message is poinient and is beautifully written. And because our site has been visited an unbelievable amount of times throughout this campaign, I’m including it below for you to read, comment, and share.
Oh – and Shire has been spending the bulk of their day visiting our site and reading your comments and posts. Perhaps Flemming will take a moment to read the thoughts below while he’s here.
Thanks, as always, for your love, care, and support.
Before I get on with the letter that Jamie and Jason Fowler hand delivered to Flemming Ornskov at their brief meeting in Chicago last Saturday, January 18 (more can be read at Jack’s new website: http://www.savejack.ca/), I am bursting with hopeful news. Due to new and close connections our MPS II community has developed with the FDA, this past week, our #SaveJack team connected directly with the FDA on one very important point. If an adverse event happened to Jack, would or could the FDA shut down the trial? This is a quote directly from the FDA this week:
‘As far as I know, and 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.’
Former FDA Commissioner Dr. Lester Crawford stated:
“The FDA, categorically, does not attach special significance to adverse events reported from such expanded access program as (one critic) has tried to join. We recognize that these programs involve less-controlled use of new drugs, and we assess the reported data accordingly. The development of a new medication is not slowed by side effects occurring outside clinical trials.”
In my eyes, this has been the only question holding us back. I can see from Shire’s perspective why they would not approve a drug for a child if it could threaten their clinical trial and shut down treatment for many other children (not including the financial burden they must look at). However, with the above answer straight from the horses mouth, there is no good reason not to submit an application to the FDA for Jack Fowler to receive Intrathecal Elaprase.
It will not harm their trial. A doctor and institution are ready. The finances are in place if there are any issues with funding. We just need Shire to say yes. With this new news, I am so so so hopeful that there is no longer anything holding Shire back from saving Jack’s life.
Please please please Flemming Ornskov and Phil Vickers, the decision has been made so easy for you!! Just say yes! And finally, here’s the letter:
Dear. Dr. Ornskov,
My name is Deb Purcell and I am the mother of a boy, Trey, who lives with MPS II. The long version of our journey is on our website at www.treypurcell.com. The short(er) version, I will share with you here.
Trey was diagnosed unexpectedly at 23 months of age on February 14, 2006. That diagnosis uprooted our lives. It changed everything about what it means to be a parent, what it means to be alive. It took my husband and I at least a year to adjust to what it meant to be parents of a child with MPS II. Most of our days were filled with tears and the only reasons we could find to live were our two children at the time, Trey and his 3-month old brother Avery.
One year after diagnosis, after a huge battle with our government to access the drug, Trey began IV Elaprase. With Elaprase giving us hope, life improved. However, as you know, 2/3 of kids with Hunter’s are cognitively affected. Beginning when Trey was two, every six months we traveled to North Carolina to have Trey’s IQ tested. With each test, we were told that the chances of Trey’s brain being affected were reduced. When Trey first tested for the Intrathecal trial at the age of five in 2009, Dr. Muenzer told us Trey’s brain would be okay. His IQ was stable and Trey was beyond the age at which his IQ would decline. When Trey was six, I asked Dr. Muenzer to test Trey one last time, to be sure. It was then we were told Trey’s brain was affected and Trey would be screened for the trial.
I cannot explain to you what it is like to live for four and a half years, wondering if my son would live or die. Because that is the difference between the severe and attenuated form of Hunter Syndrome to a parent. Attenuated means college, jobs, traveling, LIFE. Possibility. Severe means g-tubes, seizures, wheelchairs, safe rooms, vegetative state, and DEATH.
For four and a half years, every moment of my life was taken up by fear. If Trey had a toilet accident, if he hit his brother, if he forgot a word, if he put his shirt or pants or shoes on backwards, if he had a tantrum, if he didn’t finish a puzzle or scribbled on paper instead of drawing, if he choked on food, if he woke in the night, I was seized by terror and the possibility that Trey’s brain was affected. Even when Trey did great, when he learned a new word, when he shared, when he learned to cut with a knife or ride a bike or put his hearing aids in, I wondered if that meant his brain was okay. For four and a half years, I lived with indescribable fear.
When we learned Trey’s brain was affected, I had the second biggest collapse of my life, the first being upon diagnosis. When I got back up after that, I had something to fight for. The Intrathecal trial.
I planned how often to call and email Dr. Muenzer so that he would know we want Trey in the trial and are dedicated and not giving up, but not so much that I would put him off. We stopped turning on any movies or TV and hired daily tutors to keep Trey’s cognition high enough long enough so he would not drop below inclusion criteria. I interviewed doctors about symptoms of hydrocephalus so I could have some indication of whether or not Trey’s intracranial pressure (ICP) would exclude him. We did anything and everything we could- I could tell you more in person- to ensure Trey would qualify for the trial.
That qualifying trip to UNC was the hardest time of my life. There was a drug that would save my son’s life, and an arbitrary number would tell us whether or not he would get it. Waiting four hours while Trey was under general anesthetic for the qualifying tests is indescribable. I have never been so close to a panic attack. For the first two hours I distracted myself with reading and practicing yoga. As we got closer to the time when Dr. Muenzer would walk in that door and give us the news, I could only sit and stare in front of me. I could not talk to my mom or husband. Every time the waiting room door opened, my stomach jumped and my heart dropped. I began to feel nauseous and sweaty and shaky and after an hour and a half of this, I thought I was going to throw up, pass out or explode (I’m not exaggerating). When Dr. Muenzer walked in and told us Trey qualified, I collapsed with relief. Trey got to live. Trey would live. I couldn’t believe it. It took me months to understand what that really meant. Trey gets to live.
Life since Trey qualified for the IT trial has changed completely. Instead of a life of fear and loss and sadness, our family is LIVING. Really living. I don’t have to wonder what skill or word Trey will lose today or push away the thought of when he will die and what song I will play at his funeral.
When Trey first started receiving IT drug, I recorded every new thing he did. This was easy to notice because Trey had stopped making big gains. He lost 25 IQ points between August 2010 when his IQ qualified him for the trial and 14 months later when he got his first dose of IT drug in October of 2011. At that point Trey was down to one-syllabyl words and 3-4 word sentences. His proprioceptive and vestibular needs led him to hit and bang anything he could get his hands on and spank any bum within his reach. His younger siblings were not safe in a room alone with him.
Now? You really need to speak with my family and those who worked with him before and after. They will cry. EVERY LAST ONE OF THEM. There have been so many changes in Trey as a result of this IT drug, I could wrote a thesis on it, but here are a few:
I can’t even count how many words are in Trey’s sentences. His language has grown so incredibly that he uses complete sentences (he used to skip small words like ‘it’ ‘as’ ‘the’), joins sentences with the word because (never did he do that before) and uses descriptive language. Trey’s speech therapist noted that before IT drug, when she tried to play a simple game with him, he would grab pieces because he couldn’t wait and throw the game when he was done because he was frustrated and didn’t know how to say he was done. About six months ago Trey threw the game and when she asked why, he said ‘Because I am done with the game and I wanted to throw the game so the pieces would go over your head.’ More recently, Trey told her he didn’t want to play so they stopped, and about two months ago, she pushed Trey even farther, telling him she wasn’t done playing so he had to wait, so he sat and waited. A month ago he kept playing until the game was over, even though he was done.
It’s small details like these that are noticeable and some I don’t even notice right away. It was random one day that I suddenly realized Trey no longer has a need to drum and hit, that he no longer smacks people’s bums.
He began to hug us. Before the trial, I could tell Trey wanted to hug, but he would lean in and back right out again. He couldn’t hug. Now we hug for hours, which as a mom told her child was going to die, is by far the best perk.
Trey is learning to swim. He is learning his letter sounds and learning to read. I never thought he would. Trey now understands one-to-one correlation with numbers and is learning to add and subtract. No matter how many times I tried, he never made this connection with numbers before.
Our family is learning ASL and for the first time Trey has the attention to sit for an hour lesson and actually attempts to form signs with his hands.
The biggest change is quality of life. Trey can be alone with his siblings, he understands not to touch sharp knives or touch hot stoves. I can tell him to go to bed and he will. For the first time, Trey is beginning to understand and make connections. When I was trying to figure out if Trey’s brain was affected or not, Melissa Hogan described ‘passing conversations’ to me and it became clear as day. When you talk to a child who is cognitively affected, it’s like you’re two cars on a highway passing each other. The connection is missed. Trey was just like that. Every day now I see new connections being made in Trey’s brain. I feel like I can see the IT Idursulfase clearing new pathways in his brain. It is so so very awesome. Trey has a progressive disease that is no longer progressive and is actually reversing itself. I keep wondering how I got so lucky and if this really is for real.
Jamie could have been me and I could have been her. Jack could have qualified for the IT trial and Trey could have been excluded. It was the flip of a coin. Chance. What would I want her to do for me if I were in her shoes? How hard would I need her to fight for my son’s life? These are questions I ask myself.
I will fight for Jack and I will fight for Trey because they are both my boys. This means fighting until Jack gets access to drug because as a parent, it is incomprehensible to me that anyone thinks another parent could give up on their child’s life. It also means fighting to get this drug approved if anyone gets in the way. If there is one thing MPS parents learn how to do better than anything else, it is to fight. We have been fighting systems, institutions, governments, schools, hospitals, insurance and pharmaceutical companies, and injustice since the day our children were diagnosed.
What would I do to get this drug approved? ANYTHING. What would you do to keep your child alive? ANYTHING. This drug has saved Trey’s life. I would do anything to get this drug approved because it is the only thing keeping Trey alive. This is not even a mild exaggeration. That some ponder its efficacy is shocking. It makes me realize that those people have no connection to families with Hunter Syndrome. This drug is a miracle.
Sincerely,
Deb Purcell
An Open Letter To Flemming Ornskov – A Passionate and Poignant Message January 28th, 2014mcfadyena
