A new drug for Spinal Muscular Atrophy

A new drug for Spinal Muscular Atrophy


a leading cause of death among children
under the age of two is a disease called spinal muscular atrophy today we spoke
with a scientist at Cold Spring Harbor Laboratory who’s been working steadily
to find a cure it can be a deadly disease that oftentimes results in
progressive motor loss of the spinal cord and as a result of that muscles can
become weak and gradually waste away it can lead to progressive weakness and
paralysis it’s called spinal muscular atrophy and
Dr. Adrian Krainer at Cold Spring Harbor Laboratory says his hard work trying to
cure it is finally paying off in collaboration with Isis
pharmaceuticals we have developed a new type of candidate drug that’s now
being tested in in so called Phase one clinical trials and it holds promise
towards a cure for this disease wondering how the drug works
it’s a targeted drug so it targets a particular gene that’s defective in
spinal muscular atrophy called survival of motor neuron there is a version of
that gene that’s present in all patients and the drug attempts to correct the way
in which that gene functions so that it produces the correct product doctors say
when a child is born or develops this disease their quality of life is heavily
impacted it can be very severe with some children dying before age two and others
having normal lifespans but severely compromised motor function about one in
six thousand newborns worldwide are born with SMA and even though there are a few
more years to go in terms of more clinical research to be done doctors
believe a cure will be released sometime in the near future in Cold Spring Harbor
Oriana D’Agostino TV 1055 news

11 Replies to “A new drug for Spinal Muscular Atrophy”

  1. Hi all, I have type 3 and started to use Creatine, Glutamine, Vitamin B12, Folic Acid and protein shake (whey and caseine isolate). Started doing exercise daily and you will not believe the amount of muscle that I've been building up.  This works to stop muscle from wasting away and I have window periods where I don't have issues balancing etc.  Stopped falling down and can turn around in bed again. Wonderful stuff!! Hope this will help someone…otherwise, hang in there, a proper cure seems to be on its way

  2. Hello ! Our daughter is diagnosed as SMA 1. She is 13 years old. Plz can you help us how can we treat our daughter ? Tej

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