The U.S. Food and Drug Administration approved the six-minute walk test (6MWT) as an acceptable primary endpoint, or goal, for a potential registration program of the investigational treatment reldesemtiv for spinal muscular ... Read more
Systemic inflammation and bacterial infections may be key in the progression of spinal muscular atrophy (SMA), according to a recent mouse study. The research, “A severe mouse model of spinal muscular ... Read more
Lifesaving therapies like Spinraza (nusinersen) and others still in trials are indeed important breakthroughs for patients with spinal muscular atrophy (SMA), but they’re still no substitute for time-honored medical strategies like ... Read more
Heart defects occurring pre-birth may contribute to cardiac pathology in spinal muscular atrophy (SMA) patients, according to a mouse study.
SMA patients primarily have loss of specialized nerve cells called ... Read more
The 2016 SMA Researcher Meeting, the largest gathering specifically dedicated to spinal muscular atrophy (SMA), set a record with 350 attendees who discussed cutting-edge research and the future of SMA patients’ needs. ... Read more
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