Celebrate Team Momentum’s 4th Birthday with Us!
In recognition of MDA Team Momentum’s fourth birthday, we rounded up four reasons this special day is worth celebrating.
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Five Questions with DMD Researcher Dean Burkin
Dean Burkin was awarded an MDA research grant to test the effects of an existing FDA-approved drug on the function of heart and skeletal muscle in a mouse model of DMD.
Five Questions with Researcher Angela Lek
Angela Lek, a postdoctoral research fellow at Boston Children sHospital, was awarded an MDA development grant totaling $180,000 over three years to use cutting-edge techniques and a novel approach to search for drug targets in FSHD.
Five Questions with DMD Researcher Feng Yue
Feng Yue, of Purdue University was awarded an MDA development grant totaling more than $175,000 over three years to evaluate the therapeutic potential of a protein called PTEN in DMD.
Participants Sought for RA101495 MG Trial
Researchers are looking for people with MG to participate in a phase 2 clinical trial, to test the experimental drug RA101495 in people with generalized MG who are positive for acetylcholine receptor autoantibodies.
Five Questions with Neuromuscular Disease Researcher James Lupski
James Lupski, who lives with Charcot-Marie-Tooth disease type 4B., was awarded an MDA research grant totaling $300,000.
Dyla, Who Lives With SMA, is Gaining Strength Thanks to Spinraza
Meet Dyla, a sweet 11- year-old who is greatly benefiting from the recently approved SMA drug, Spinraza.
Simply Stated: What’s a Biomarker?
You’ve heard the term “biomarker,” but what exactly is a biomarker and why does it matter in regard to neuromuscular disease? Learn more here:
Solid Biosciences Launches DMD Gene Therapy Trial
Solid Biosciences announced the launch of its first clinical trial for SGT-001. The phase 1/2 study, called IGNITE DMD, is designed to assess safety and efficacy of a single dose of SGT-001 in children and adolescents with DMD.
Encouraging Results for Firdapse in LEMS Trial
Catalyst Pharmaceuticals has reported encouraging results from its second phase 3 clinical trial to test the investigational drug amifampridine phosphate (brand name Firdapse) in people with LEMS.
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