After significant collaborative work among many stakeholders in the spinal muscular atrophy (SMA) community, we are pleased to share that the Secretary of Health and Human Services (HHS), Dr. Alex Azar, has accepted the recommendation to add SMA to the Recommended Uniform Screening Panel (RUSP) for newborns. This is a landmark decision for the SMA . . .
The Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), a federal committee that oversees newborn screening, voted today to add spinal muscular atrophy (SMA) to the national list of disorders recommended for screening at birth. Following today’s vote, the Secretary of Health and Human Services (HHS) will make a final decision on whether . . .
Kevan Chandler, who lives with SMA and traveled through Europe in 2016 in a backpack, is heading to China. Along the way, he hopes to inspire others and “demonstrate that life is full of possibility and hope.”
In MDA’s pursuit to find treatments and cures for neuromuscular diseases we awarded four grants to researchers studying SBMA and SMA in 2017. Check out their research here:
Former MDA National Ambassador Joe Akmakjian shares his tips and tricks for a successful moving experience.
As we celebrate the new year, former MDA National Ambassador Joe Akmakjian takes the time to celebrate accomplishments of the past while looking forward to the opportunities that lie ahead.
Researchers are looking for individuals with SMA type 2 to participate in the phase 1 STRONG clinical trial, to test safety, dosing and proof of concept for efficacy for its SMA gene replacement therapy AVXS-101.
Meet Dyla, a sweet 11- year-old who is greatly benefiting from the recently approved SMA drug, Spinraza.
Faith Fortenberry, who lives with SMA, is getting stronger everyday thanks to Spinraza.
The Cochran’s, whose daughter Lindsay lives with SMA, share their experiences providing care to a loved one who lives with a NMD.
