2019 Clinical & Scientific Conference Highlight: What’s New in Neuromuscular Diseases?

At the 2019 MDA Clinical & Scientific Conference, one scientific session of interest centered on what’s new in neuromuscular diseases (NMDs). In this session, presenting scientists discussed progress in therapy development and clinical trials for a range of NMDs, including Becker muscular dystrophy (BMD), congenital muscular dystrophy (CMD), congenital myasthenic syndromes (CMS), myotonic dystrophy (DM), . . .

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Encouraging Results in Phase 1 CALLISTO Trial to Test Omigapil in CMD

A phase 1 trial to test omigapil in congenital muscular dystrophy (CMD) conducted at the National Institutes of Health is complete and data indicate it met its primary objective. Results from the trial, also known as CALLISTO, show a favorable pharmacokinetic profile of omigapil and demonstrated that the drug was safe and well-tolerated in children and . . .

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Meet MDA’s Newest National Ambassador Justin Moy

Justin Moy’s future is brimming with potential. The 17-year-old Concord, Mass., native is currently a senior at Concord-Carlisle High School, where he enjoys science and participating in the school choir. Although he hasn’t yet figured out what college he’s going to attend, he knows what he wants to do once he gets there. “I want . . .

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“Such a Small World”: Top Team Momentum Fundraising Team Fueled by Lifelong Friends

“My husband and I always tell people this because it is the truth: Henry is our hero,” says Stephanie Betts, an Team Momentum runner and proud mother of son Henry who lives with congenital muscular dystrophy. “He is incredibly strong. Our quote on the back of our Henry’s Heroes shirts states, ‘True strength is in the soul and spirit, not in muscles.’ He is an amazing kid who has the best attitude that just draws people in. We are so incredibly lucky to be his parents.”

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