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Melanie’s Story of Strength
"You are so much more than your condition!"
1st National LGMD Conference
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Abby’s Story of Strength
“I know my weakness doesn’t define me…”
- This Back-to-School Season, MDA National Ambassador Justin Moy Looks Ahead to Sophomore Year August 20, 2019School creeps up on you! At first you look at your three-and-a-half months on the calendar and marvel at the expansive possibilities, but here I am three months later and I’ve done maybe three quarters of what I wanted to get done. It’s not to say that I’ve been lounging around, although there has been […]
- Faith Goes Camping: Our National Ambassador Attends Her First MDA Summer Camp August 16, 2019Reagan Imhoff, 14, is a former MDA National Ambassador (2014-2015) and a current camper at MDA Summer Camp in Wisconsin. Reagan, who lives with spinal muscular atrophy (SMA), thoroughly enjoys MDA Summer Camp, and she was excited to connect with current MDA National Ambassador Faith and share perspectives on what it’s like to stay at […]
- Simply Stated: What is Spinal Muscular Atrophy? August 15, 2019Spinal muscular atrophy (SMA) is a neuromuscular disease that affects and impairs voluntary muscle movement. It is the leading genetic cause of infant death. SMA is caused by a mutated or missing survival motor neuron 1 gene (SMN1) that prevents the body from making enough survival motor neuron protein (SMN), ultimately resulting in motor neuron […]
- The ALS Association, ALS Finding A Cure, and MDA Co-Fund Clinical Trial Grant to Study Restoring Immune System Function to Treat ALS August 9, 2019The ALS Association, ALS Finding a Cure (ALSFAC), and MDA announced they have jointly awarded a clinical trial grant totaling more than $2.5 million over two-and-a-half years to leading investigators at the Houston Methodist Neurological Institute and Massachusetts General Hospital. The principal investigator is Stanley Appel, MD, co-director of Houston Methodist Neurological Institute, chair of […]
- Scholar Rock Announces Positive Results from Phase 1 Study of SRK-015 to Treat SMA August 7, 2019Scholar Rock Holding Corporation announced positive results from its phase 1 clinical trial evaluating SRK-015, a therapy for treating spinal muscular atrophy (SMA), at the Cure SMA Annual Conference held June 28 through July 1 this year. Consistent with previously announced interim findings in February, the final results showed positive safety, pharmacodynamic, and pharmacokinetic data in healthy volunteers, supporting […]