All posts by mcfadyena

06 Oct 2015
Sticky Post By Posted in Advocacy, Blog Postings, Media, Morquio, MPS IVA, Newspaper, Saskatoon Siblings Permalink

Province says no to medicine for three children, but will review decision

Sticky Post By On 6 October, 2015

Children suffering from crippling genetic disease
CBC News Posted: Oct 05, 2015 6:43 PM CT Last Updated: Oct 05, 2015 7:00 PM CT
The province’s health ministry says it won’t pay for a costly drug that may help three children with a crippling genetic disease, but Health Minister Dustin Duncan said he will ask for a second opinion.

Muhammad Abdullah, 12, Khadija Amir, 10 and Sara Amir, 8 all have a rare enzyme deficiency called Morquio A Syndrome (also called MPS IV type A).

Without the missing enzyme, cellular waste builds up in the bones, tissues, organs and muscles.

The condition is stunting the children’s growth, twisting their joints, affecting their eyesight and hearing, and making it tough for them to breathe. Two of them use wheelchairs at school.

Muhammad Amir Akhter is the children’s father. He said the disease can be life-threatening.

“Enzyme replacement therapy like Vimizim is the last resort,” Akhter said.

He said despite the current decision, he is staying positive. “It’s pretty hard for us, because this is the last hope here.”

Health minister will get a second opinion

Health Minister Dustin Duncan explained the decision to reject the drug coverage.

“These are very, very complex cases,” said Duncan.

He said in this case there is not enough evidence to prove that the drug will be effective for people over the age of five.

“There’s a lot of opinions on this. And so while we do rely and have relied on a previous case, on an out-of-province expert I’m asking the ministry to consult with others that may have some information on this particular case.”

Duncan said the ministry will review some of the medical debate about the efficacy of the drug, and will invite the Akhter family to submit any further evidence they find.

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

The Opposition NDP said the children should get the drug since it is their only hope. It added that effectiveness can be monitored while they take it.

05 Oct 2015
Sticky Post By Posted in Advocacy, Blog Postings, Media, Morquio, MPS IVA, Saskatoon Siblings Permalink

LIFE-SAVING TREATMENT DENIED FOR AILING SASKATOON SIBLINGS

Sticky Post By On 5 October, 2015

The Saskatchewan Ministry of Health has denied funding for a life-saving treatment required by three siblings from Saskatoon.  8 year-old Sara Amir, along with her siblings Khadija, 10 and Muhammad, 12, suffer from MPS IVA (Morquio Syndrome) and require the life-saving treatment immediately in order to halt further progression of their devastating disease. The Isaac Foundation and The Canadian Society for Mucopolysaccharide & Related Diseases (Canadian MPS Society) are calling on Health Minister Dustin Duncan and Premier Brad Wall to take action, review and reverse the decision, and ensure that treatment begins immediately. Without access to therapy, Sara, Khadija, and Muhammad face a long and painful decline and death at a young age. The Isaac Foundation and the Canadian MPS Society will meet directly with Minister Duncan at 11:00 am on Monday, October 05th to urge the Minister to intervene and save the children.

Sufferers of Morquio Syndrome lack an enzyme in their blood that breaks down cellular waste in the body. This waste builds up in the bones, tissues, organs, and muscles of affected individuals and leads to many devastating symptoms including heart and airway disease, corneal clouding, stiffening of the joints, shortened stature, and premature death.

Andrew McFadyen, Executive Director of The Isaac Foundation, an advocacy, research, and family support organization that specializes in MPS related diseases, is disappointed by the decision and is urging an immediate reversal to save the lives of the children. “The decision to deny this life-saving treatment for these children is outrageous, heartless, and cruel. This therapy has been recommended by a Canadian Expert Panel comprising of MPS experts from BC, Alberta, Saskatchewan, Ontario, and Quebec, and International Treatment Guidelines for Morquio Syndrome highlight this treatment as the gold standard of care for affected patients. To prevent these kids from receiving the care they require is needless and callous, and shows a reckless disregard for what our Canadian Health Care System purports to stand for –a health care system that was ironically born in this very Province.” He adds, “The Ministry is telling this family to enter a long-term palliative approach to care for their children instead of ensuring that disease progression is halted now. They are allowing them to die, instead of taking leadership and doing the responsible and ethical thing by providing them with the help they need. It’s shamefully disgraceful.”

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

McFadyen is puzzled as to why access for treatment was denied, especially considering the weight of available evidence and Canadian and International expert guidelines urging it’s approval. In addition, the Province already provides access to patients suffering from every other form of MPS requiring the same kind of treatment. He expects it comes down to financial considerations due to the high cost of the drug, coupled with a recent negative recommendation from the Common Drug Review (CDR), that has clouded the judgment of decision makers at the Ministry.

Jamie Myrah, Executive Director of the Canadian MPS Society, a national patient association that serves those affected by MPS and related lysosomal diseases, couldn’t agree more.   “We know this treatment works and the Province knows that ERT’s for MPS diseases are currently the only chance patients have of living long, healthy lives. We recognize that the CDR gave this treatment a negative recommendation, but it did so for all the other MPS ERT’s as well; yet every province in the country – including Saskatchewan – still ensures access for those patients. While ERT treatment for MPS is expensive, it is often just as expensive – if not more expensive – to not treat these kids. It simply doesn’t make sense to make these children suffer a slow and painful decline when we could stop or dramatically slow the progression of the disease with the treatment being requested.”

Naturally, the Akhter family has been devastated by the diagnosis of their kids. “Since receiving these diagnoses, we’ve been sick with guilt, grief, and worry. Our world will never be the same,”said the siblings’ father, Amir. “The only comforting factor since receiving this news has been the knowledge that a treatment is available to help my kids.  We want to be able to do everything we can for our children to give them everything they deserve in this world, including a fair shot at a healthy life. We’ve now lost hope –our kids have been handed a death sentence by this government. This disease is not their fault.”

McFadyen questions the process used by the Saskatchewan Ministry of Health in making their decision, noting the family first requested access in March of 2014. He notes that in August of 2015, the file was finally sent to Ontario for review by one individual alone, with no opportunity allowed for a rigorous discussion with the rest of the MPS Experts throughout Canada –experts that deal with the disease on a daily basis and some of whom have seen first-hand how well other children receiving the treatment are doing. “The only thing stopping these children from beginning their treatments is the lack of funding by the Province. It’s incredulous to me that one individual gets to pick and choose who does or doesn’t get access to the life-saving treatment they need, especially considering the fact that a precedent has been set with other children in this province and in other provinces across the country receiving access to the medication. One individual is making life-altering decisions on behalf of this Ministry and the people of Saskatchewan, and it’s led to these kids lives being left in peril.”

He is hopeful that a review and a reversal of this decision can take place immediately, and both he and Myrah are urging the Health Minister and the Premier to take action now. Says McFadyen, “Brad Wall’s government has the opportunity to do the right thing and ensure that Sara, Khadija, and Muhammad receive the treatment they so desperately need. It’s the role of government to protect and ensure fair and equitable access to Health Care for all people in Saskatchewan, regardless of whether they are impacted by a rare disease or not. This family needs to concentrate on treating their children’s disease, not wading through a bureaucratic maze of paperwork and pinning their hopes on the appeals process or the recommendation of one person alone –a person who has never used this therapy or seen it’s effectiveness – at the Ministry of Health. We strongly call on this government to take action and save the life of these children now. They can’t afford to wait.”   

                                                                                                                         # # #

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

15 Sep 2015
Sticky Post By Posted in Advocacy, Blog Postings, Media, Morquio, MPS IVA, Newspaper, Saskatoon Siblings Permalink

Saskatoon family awaits answer on treatment for crippling disease

Sticky Post By On 15 September, 2015

Three children all suffer from genetic enzyme deficiency that could lead to early death
CBC News Posted: Sep 15, 2015 5:30 AM CT Last Updated: Sep 15, 2015 6:43 AM CT

A family in Saskatoon is waiting and hoping for the solution to a triple heartbreak. Three of the five children suffer from a crippling genetic disease that could kill them.

Muhammad Abdullah, 12, Khadija Amir, 10 and Sara Amir, 8 all have a rare enzyme deficiency called Morquio A Syndrome (also called MPS IV type A).

“Until the age of three to four nobody can judge that there’s anything wrong with them actually,” their father, Muhammad Amir Akhter said. “After that the symptoms appear, and almost every part of the body is affected.”

Without the missing enzyme, cellular waste builds up in the bones, tissues, organs and muscles.

The condition is stunting the children’s growth, twisting their joints, affecting their eyesight and hearing, and making it tough for them to breathe. Two of them use wheelchairs at school.

It’s so rare, it only appears in an estimated one out of every 200,000 to 300,000 children.

Hope placed in costly synthetic enzyme

There is no cure. But a synthetic enzyme called Vimizim could slow or even halt the progression of their disease, and help them live longer.

An advocacy group called the Isaac Foundation said Health Canada approved Vimizim more than a year ago.

Now Akhter and his wife Shazia Amir are waiting to find out if the province will pay the cost, which could be in the hundreds of thousands of dollars each year.

“It’s really hard to wait for the decision,” Akhter said. “You know we are anxiously looking forward to see every day morning, we are looking for any positive response from the government side to get that too, to get started that replacement therapy.”

Health ministry considering compassionate coverage

He said they applied seven months ago. However, Saskatchewan’s Ministry of Health said it only got complete information from the children’s doctor late last month.

It also said the national Common Drug Review made a “Do Not List” recommendation for Vimizim for clinical reasons.

However, the ministry is considering compassionate coverage on a case-by-case basis.

In the case of Akhter’s children, the ministry has sent their requests to an out-of-province specialist for review and advice.

If they are turned down, Akhter said he will consider moving the family to another province that is willing to pay for their treatment. The Isaac Foundation said Ontario and Quebec have reimbursed patients for Vimizim.

14 Sep 2015
Sticky Post By Posted in Advocacy, Blog Postings, Media, Morquio, MPS IVA, Saskatoon Siblings Permalink

For Immediate Release – Saskatoon Siblings Await Funding For Life-Saving Treatment

Sticky Post By On 14 September, 2015

FOR IMMEDIATE RELEASE

SASKATCHEWAN SIBLINGS AWAIT FUNDING FOR LIFE-SAVING TREATMENT

Children Diagnosed With Ultra-Rare Condition; Treatment Already Being Funded In Ontario and Quebec

(Sept. 14, 2015) The Saskatchewan Ministry of Health is currently considering an application for exceptional funding of a life-sustaining treatment required by three siblings from Saskatoon. 8 year-old Sara Amir, along with her siblings Khadija, 10 and Muhammad, 12 have been diagnosed with Morquio Syndrome, and all three require the life-sustaining treatment immediately in order to halt further progression of this devastating disease. The Saskatchewan Ministry of Health received their application to begin treatment with Vimizim, an enzyme replacement therapy approved by Health Canada in July 2014, seven months ago. A review of the application has just been initiated and a decision is expected in the coming weeks.

The children suffer from a rare enzyme deficiency called MPS IVA (also known as Morquio A Syndrome). Sufferers of Morquio Syndrome lack an enzyme in their blood that breaks down cellular waste in the body called glycosaminoglycan (GAG). These GAGs build up in the bones, tissues, organs, and muscles of affected individuals and lead to many devastating symptoms including heart and airway disease, corneal clouding, impaired mobility, shortened stature, and premature death.

While there is no known cure for Morquio Syndrome, a treatment does exist. Vimizim is an Enzyme-Replacement Therapy (ERT) designed to provide patients with a synthetic version of the enzyme they are lacking by infusing small doses into the patient’s bloodstream on a weekly basis. The treatment slows down or halts the progression of the disease in patients, improves endurance, walking distance, breathing problems, and provides other benefits to sufferers that dramatically improve their quality and length of life. International experts and a Canadian Panel of Genetics Specialists have all recommended Vimizim as the front-line treatment for Morquio Syndrome. It was approved by Health Canada in July 2014, and has been reimbursed for use by patients in Ontario and Quebec. Recently, the National Institute for Health and Care Excellence (NICE) recommended reimbursement for all patients suffering from Morquio Syndrome throughout the UK.

Andrew McFadyen, Executive Director of The Isaac Foundation, an advocacy, research, and family support organization that specializes in MPS related diseases, is urging Health Minister Dustin Duncan and Premier Brad Wall to expedite their decision so the children can get the immediate help they need. Since the application was submitted in March, McFadyen has met with Minister Duncan twice to present all of the expert opinion and Canadian and International data that exists regarding the treatment. “Minister Duncan has been privy to all opinions and guidance from the best medical minds across the globe. All of them have recommended this treatment for these kids and I’m confident that a positive decision will be returned in a speedy fashion.”

Though hopeful of a positive outcome for the family, McFadyen can’t help but feel frustrated by the length of time it’s taken to render a decision. “The Minister has had this file for over 7 months, and the initial request for therapy was submitted 19 months ago. Saskatchewan already pays for every other available treatment for MPS Diseases – MPS I, MPS II, MPS VI. This treatment does the exact same thing – saves lives. With the UK and the US already ensuring access to this treatment, there is ample evidence available to help them with their decision. We’ve given them everything they need – it’s time to take action and save these kids.”

Jamie Myrah, Executive Director of the Canadian MPS Society, a national patient association that serves those affected by MPS and related lysosomal diseases, couldn’t agree more. “The Canadian Expert Panel and the International Guidelines for treating Morquio A Syndrome both call for treatment to begin as soon as possible to stave off the devastating effects of this disease. With every day that passes, the chance that irreversible symptoms will appear increases. I am hopeful that the Saskatchewan government won’t allow bureaucracy to have a negative impact of the lives of these children and am therefore confident that a positive decision will be returned soon.”

Myrah and McFadyen both note how impactful the treatment has been for patients already receiving therapy in Canada and in most other developed countries throughout the world. Myrah states, “We are seeing kids improve dramatically because of this treatment – kids’ internal organs reducing back down to a normal size, rates of growth increasing, heart function improved, walking distance increased and the use of mobility aids reduced. Until recently, only supportive care that treats the symptoms of the disease was available to patients, including medication, multiple surgeries, and ongoing occupational and physical therapy. By delaying access to the first and only pharmaceutical treatment option available, governments are leaving patients dependent on supportive therapies that do not address the underlying cause of this severely debilitating disorder. We know this works and we know it changes lives.”

McFadyen adds “We have a video of a 17 year old girl prior to starting treatment and she can’t walk more than 10 steps without having to stop. She’s in agony and it’s heartbreaking to see. After 12 weeks on therapy, she’s happy and walking long distances normally. I’m hopeful we can ensure the same opportunity is given to the Akhter children. It’s the role of government to protect and ensure fair and equitable access to Health Care for all Canadians –regardless of whether they are suffering from a rare disease or not – and we’re calling on this government to take action and save the lives of these children now. They can’t afford to wait.”

 # # #

For more information about this topic, or to schedule an interview to discuss, please call Andrew at 613-328-9136 or email Andrew at mcfadyena@me.com.

 

13 Aug 2015
Sticky Post By Posted in Blog Postings Permalink

Saying Goodbye Has Never Been More Difficult

Sticky Post By On 13 August, 2015

Everything comes to an end in this world, but sometimes we wish we could have a bit more time before the special things in life do.  If we could, we’d make time to stand still for a short while, just long enough that we could pause and enjoy a special memory, a special person, a special moment, for just a little longer.  Today was one of those days.

Dr. Julian Raiman came into visit with Isaac this morning for one last time before he heads back home to the UK to continue his stellar career in his home country.  He dropped in for his last visit with Isaac, his last check-up, his last report taking, his last few moments caring for our son.  It was a difficult time for us as parents and our kids, and I didn’t want the visit to end because, when it did, our time together would officially be over.

Julian has been with Isaac and our family for over 10 years.  He has grieved with us as he’s given us bad news, cried with us as we’ve adjusted to new circumstances in life, and celebrated with us during health improvements and triumphs.  More importantly, he’s guided us along this journey – always at the forefront, always in the lead, and always assuring us that we still had Hope for our son.

There aren’t many people I trust with my son’s life, and Julian Raiman has stood out amongst those that I do.  His kind and caring nature shine through when he is with our kids and his loving demeanour has always made them feel safe and secure in the hospital, especially when things have been difficult.

His compassion always allowed him to connect with our rare disease families, and his empathy allowed him to grieve alongside us.   He understands us and recognizes how difficult these journeys are.  At the same time, he always gives families the strength to keep fighting, encouragement and hope that will sorely be missed by us all.

Julian has also led the charge for our children to get access to expensive treatments – treatments that save lives.  He’s traveled around the world presenting to patient groups, organizations, and government officials.

image2-2Julian has led clinical trials and helped pioneer new treatments for our kids.  I was with him at a Denver Patient Symposium recently and he passionately told the crowd that “It’s nice when we have medicines that make us get better.  But it’s just as important to have medicines that prevent us from getting worse.”  To me, that is at the heart of every battle I’ve fought throughout the past decade for our kids, and Julian eloquently summed up the need for us to have access to treatments for our kids in one powerful sentence.  He’s like that – inspirational and uplifting, yet modest and humble at the same time.

Julian has championed our childrens’ causes, he’s tirelessly joined our battles.

He’s been consoling and impactful.  And has made the world a better place for us all.

Julian has been a life changer and a life saver.

And Julian will be greatly missed.

Farewell, Dr. Raiman.  Thank you for all you’ve done for my son, our family, and all the families that The Isaac Foundation has been lucky enough to be involved with.  You are a true hero to us all.

With Love,

The Isaac Foundation

 

23 May 2015
Sticky Post By Posted in Blog Postings, Jack Fowler Permalink

Race Weekend – For Jack

Sticky Post By On 23 May, 2015
Well – race weekend is here and we’ve all arrived in Ottawa to Run for a Reason. We’re here to find a cure for MPS. I’ve done more training than any other race I’ve been in, and have been running for a lot of different kids during that time. It’s our brave kids that keep me going – both in my drive to find a cure and while I’m out there trying to reach the finish line.

In everything I do, I have my son Isaac as my inspiration. And this race is no different – race day is for him. But I’m also running for another little boy, someone I love dearly, but someone I can’t help but feel I’ve let down. I’m running for the one boy that I haven’t been able to win the battle for, the boy who keeps me up at night with worry, sorrow, and guilt. I’m running for you, Jack Fowler, with a heavy heart but with resolve to keep fighting for you; with a promise to do whatever it takes to help you win your battle.

We’ve been lucky to play a small role in the lives of many kids and families during the past 9 years – something I’ll always be proud of. We’re closing in on $1 million donated to research projects aiming to find a cure. We think we’re close to a cure for MPS VI, and the incredible MPS II Research Fund is currently funding 3 promising Gene Therapy projects. We’ve taken on drug companies on behalf of our kids, we’ve won battles big and small. But for Jack, we continue to come up short and hit barrier after barrier – barriers I’m finding difficult to break through, jump over, or move around.

I feel the weight of this inability to help Jack more than ever, and I feel like I’ve let him and his family down. When I first got involved in the fight to save Jack, I promised I would do everything in my power to get him the treatment he needs. We met with Shire, launched public campaigns, hit the media circuit, and shared his story around the world. Today, a year and a half later, we still haven’t found the right way to help, and I’m heartbroken that we haven’t been able to access treatment yet.

Oftentimes, parents connect with me as a last hope – the last person to turn to in their battle to get treatment for their kids. And I’m always here to help. But so far we’ve failed, and I feel guilt and shame, broken and hurt. And I feel it all strongly because if I can feel proud for those that we’ve been able to help, I should also accept the pain that comes from those that haven’t been successful in helping yet.

For Jack, like other kids, I’ve always felt like I was the last hope – if everything we do isn’t successful, then there would be nowhere else to turn. But I’m wrong – I’m not the last person that can help. That last hope doesn’t sit with me. The last person that can help is Flemming Ornskov, CEO of Shire PLC – the drug company that makes the treatment Jack so desperately needs. Flemming continues to act as judge, jury, and executioner, and he truly is the only person who can step in and make a difference.

So this run is also for you, Flemming. Dedicated to you as a final plea to find it within yourself to be brave – to go out and help those you purport to care about most as a company. This run is for you, Flemmming, in hopes that you see that this guilt I bear should be shared by you – that your legacy as a person and a businessman will always come back to this case – to this little boy – and the decisions you make about his future. It’s for you, and for Jack, with a promise to you both that I won’t ever quit the fight to save him, no matter what road that battle may take me down.

You’ll be with me this weekend, Jack, like you’re with me each and every minute of every day. You inspire me to work harder, fight stronger, to never give up. I promise I’ll run to the end of the road for you, and beyond.

#Hope  #SaveJack

If anyone out there would like to support our Run – our Hope for a cure – please click through and spare what you can.
http://www.gifttool.com/athon/MyFundraisingPage?ID=2012&AID=2997&PID=497389

 — with Jamie Lynn-Brooks Fowler, Deb Cehak Purcell and Kristin Higginson.

17 May 2015
Sticky Post By Posted in Blog Postings Permalink

Run For A Cure – Training

Sticky Post By On 17 May, 2015

It’s 6 days until race day, and I was starting to feel poorly that my official run next week wasn’t going to be dedicated to our MPS VI crew. So – I rectified that this morning and did a 1/2 marathon for training! This one is for all the MPS VI kids I’ve been lucky enough to help in this country (Isaac, Jasper, Violet, Aleena, and Kamie) and for the incredible kids I’ve been lucky enough to follow along and watch their journey from the start (Lillian and Holden). This run is also for the MPS VI families I’ve been fortunate to know online, to the families who have helped us along with whatever battle we’ve been fighting (Ethan and Frida). And finally, this one is for you Kendra – a hero who led the way for our kids to get the treatment they need approved during the clinical trials.

Today’s run was a good one to dedicate to my fellow MPS VI families – it’s the first one since I returned back from New Orleans, the first on since we received our update on the Gene Therapy project we’ve given over $1/4 million towards and get it off the ground. I’m still waiting for permission to share the news we received on where things stand, but what I can say is that I truly believe we’ve moved on from HOPE to HERE. Everything we’ve worked so hard to see happen is now upon us, and I’m waiting with anticipation to share everything with you as soon as I can!

Working to bring ERT to Canada for our kids was tough. I lost a lot of sleep fighting for Isaac, Jasper, Violet, Aleena, and Kamie, and I lost a lot of time away from my kids during the busiest moments of those battles. I’m so very lucky to have played a small role in helping you all have the chance at life you deserve. Knowing you these kids has changed my life, and I’m a better person for it.

At the same time, I’ll never forget the moment I had to tell Isaac I couldn’t spend any time with him – time I had promised was his – because I was working on a media campaign to help Aleena get the treatment she needed. Isaac looked me in the eyes, ran over and hugged me and said “It’s OK, Daddy. You’re working to save her like you did me and I understand.” It was one of the most heartbreaking and sweetest moments of our time together, but it made me realize that I needed to make some changes in my life and ensure that I could do both. It’s one of the things that helped me make the final decision to leave my classroom and focus full time on finding a cure for our kids because if we find that cure, all of our battles will be over.

And I think we’re close. No – I KNOW we’re close. And thinking of all of you during my run today made it easy out there, even though I was running through our cottage roads and up and down hills for the majority of my time running.

Our RUN FOR A REASON event has raised over $300,000 over the past 10 years – money that we’ve sent directly toward finding a cure for our kids. It’s fitting that this 10th anniversary run is one that takes place while we’re on the cusp of finding that cure for our kids, and I can’t wait to get out there next weekend and celebrate on the course.

I’m incredibly blessed to have all of you in my life. Thank you for enriching my life the way you have. I wouldn’t be the same without you.

If anyone out there would like to support our Run – our Hope for a cure – please click through and spare what you can.
http://www.gifttool.com/athon/MyFundraisingPage…

13 May 2015
Sticky Post By Posted in Blog Postings Permalink

Run For A Cure – For Ellen

Sticky Post By On 13 May, 2015

We’ve run a lot of places together, Ellen and I, and I can’t imagine a better partner to be at my side on this long road than you.

Our latest journey has taken us to New Orleans, to the American Society of Gene Therapy Conference, as we search for a cure for our son and all the other families battling MPS throughout the world. This training run took me through the streets of NOLA, out past the insanity that is Bourbon Street and through the historic French Quarter. It started out as a short run, but kept getting longer and longer as I thought about the significance of our trip here – someone at this conference holds the future of our son in their mind, in their lab. We need to find them, fund them, and make our cure a reality.

Ellen is my partner in life, my best friend, the mother of my kids, and the strongest person I’ve ever known. She’s the reason why our Foundation has grown, why we’ve been able to achieve what we have and help the kids we’ve been so lucky to help. She’s the person I laugh with, cry with, grieve with, and the only person I can talk to about anything.

It sometimes gets hard to talk about my own son with other families – I’m still coping with diagnosis, still in disbelief that this is where our life has taken us. But running The Isaac Foundation sometimes makes me feel I have to be strong for the other families out there, which usually means I hide the fear I have inside about where my own journey with MPS will take me. I’m lucky to have Ellen with me to talk to, share those fears, and to help me see how close we are to winning our fight.

I’d like to say that my final run on Race Day is for Ellen, but that race and that run is reserved for someone else. She’ll have to be content with this run being for her – the one through the streets of New Orleans while we are on our latest journey together – an important one toward finding the cure for our son.
I love you, Ellen. More than ever before.

If anyone out there would like to support our Run – our Hope for a cure – please click through and spare what you can.
http://www.gifttool.com/athon/MyFundraisingPage…

08 May 2015
Sticky Post By Posted in Blog Postings Permalink

Run For a Cure – Training Run For Deb

Sticky Post By On 8 May, 2015

This long run is for you Deb, and for you, Trey. 16K is a long training run, but thinking of your family and thinking of the journey we’ve been on together got me through it all. Our kids were diagnosed at the same time, and you were the first family we met after that diagnosis took us down paths we never knew we would take. I’ve watched you worry over Trey, cope with horrific news, and then fight like hell to change the world for him.

And you have. And I couldn’t be more proud to know you; I couldn’t be more proud to have you on our team, side by side.

Much like our battle with MPS, my run today felt like every step was uphill. It felt like I would never make it to the end. But also like MPS, I know we’re near the top of that hill, and the run back down toward the finish line will be easier and faster. We’ll get to that finish line for our kids. We’ll get there soon, and we’ll get there together.

Thanks for continuing to inspire us. Send my love to your family.

If anyone out there would like to support our Run – our Hope for a cure – please click through and spare what you can.
http://www.gifttool.com/athon/MyFundraisingPage…