Marco, 21 now, is confined to a wheelchair, unable to stand, and on a breathing machine. Alexander, my son is 14 years old, will have the same fate.....
Imagine living with no treatment , no hope and knowing the condition is so rare that there is no incentive for medical research!!
Alexander's story .....
Alexander is a beautiful boy who came into my life a healthy, robust and perfect child. His arrival made our family complete. We were so happy. When he was a toddler, I used to call him Hercules!
Alexander had it all. He was smart, advanced in school, good at sports and musically talented. He was admired by his classmates and he had lots of friends.
But, as the years passed, when he was a bout 8 years-old, we noticed that he was changing. We did not understand why he was loosing so much weight, despite a healthy diet. Soon he started to walk slightly on his toes and this was peculiar.
We went from doctor to doctor, from small hospitals to big ones for several years after, trying to find out why! It took almost 4 years to get an accurate diagnosis.
A little over a year ago, in 2016, on my birthday, just before mother's day, we received the diagnosis over the phone. A rare, severe, deadly and rapidly progressive type of muscular dystrophy (MFM6). This genetic condition happened spontaneously and is not carried in our family.
It was devastating! How could this happen? My husband and I literally fell to our knees. A dreadful day, no one can even imagine! Our beautifull, healthy boy ... Why? It felt like a death sentence.
We deperately searched for information everywhere. On the internet, facebook, medical journals, anywhere we could think of.
Finally, we found a you-tube video about a boy with the genentic mutation BAG3 P209L, streaming across the screen. A beautiful boy like my son Alexander, his name ... Marco Vignale. During sleepless nights, we googled, searched on facebook, and even white pages. It is on white pages we finally found that Marco lived in California.
We were also able to locate one scientist, Dr. Monte Willis, at the University of North Carolina, who had done some basic work with this mutation, but had no idea that children with this condition were still alive. All the known children with this disease, died within less then 5 years from diagnosis. There were at least 15 children killed by this disese since 2009. We told him about Marco. We continue to colaborate and plan how to advance research. We have personally sponsored his work.
We have estabilished a Charity organization devoted entirely to research; Alexanders's Way Reserch Fund, inc. Donations to this charity are tax deductible. You can find this charity at www.alexandersway.org You can make tax deductible donations directly to our charity. The charity will give hope to all other children who got unlucky enough to get this genetic mutation. We are publically advocating for all children affected, now and in the future.
Meanwhile, Alexander rapidly declined. He lost his ability to run, he could no longer play sports and soon after, he could not longer walk. This year he required foot surgery to both his feet. His skeletal muscles are wasting and his heart is starting to be affected.
Imaginne living with such a rapidly progressive condition, with no hope or treatment. We know that there are others with this condition, and we are trying to locate them.
Alexander is recovering slowly. He is walking again, goes to school, plays his music still, and tries to be happy and make the best of it.
But, this disease is relentless, it is inching its way further each day, and eventually will strike. Marco is in much worse condition then Alexander is now. This disease is predictable in its cruelty and deadliness. The other known children are gone, all within 5 years. My heart goes to their families that carry the burden of grief after such a devastation and loss.
Since then, we have located other scientists that can develop the treatments needed to save our sons, as well as others with this disease and other similar conditions.
Cutting Edge New Research!
Dr. Willis at University of North Carolina, is working on a drug to reverse the effcts of this disease.
We are in negotiation with another expert in gene therapy. He has done amazing work with a similar genetic mutations.
There are no government funds for the groundwork for a treatment for such a rare disease, and no pharmaceutical company will invest in it at this stage. Once we show progress we may be able to attract pharmaceutical companies. Without your donations we can not succed.
My son, Alexander, and Marco, are like any other children. They dream of the future and hope. Please help us restore their dreams before they are lost. They have already suffered more than you and I can imagine, but they keep on going, with smiles on their faces.
No one this young should suffer, and have to face their mortality. More importantly, they must feel that the world is good, and on their side.
We have a limited time!!
Please help them fight and win!
We pray and hope for your support.
Please help us!
Laura Zah and Stephen Greenspan
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