Moore suffers from spinal muscular atrophy (SMA), a disease that affects one in 10,000 Americans. “It’s a neuromuscular disease, so it affects all the muscles in the body, except really the ...

Adult-onset spinal muscular atrophy (SMA) is a rare neuromuscular disorder that primarily affects the motor neurons in the spinal cord, leading to muscle weakness and atrophy. SMA is part of a ...

Subjects receiving the therapy demonstrated clinically meaningful enhancements in physical and muscle function.

A Co Down woman is encouraging people living with a muscle wasting or weakening condition, their families and carers to join ...

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude Children's Research Hospital led the first in utero treatment of SMA ...

INFANTILE progressive spinal muscular atrophy (Werdnig–Hoffmann syndrome ... The mother, a healthy 29-year-old Negro woman, had had an uneventful pregnancy except for minimal hypertension ...

Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for ...

Whether it's investigating the financials of Elon Musk's pro-Trump PAC or producing our latest documentary, 'The A Word', which shines a light on the American women fighting for reproductive ...

05. Nearly six years ago, the Food and Drug Administration approved Zolgensma, a Novartis gene therapy for the fatal neuron-wasting disease spinal muscular atrophy. It heralded a new era of ...

Scholar Rock contends its experimental drug for spinal muscular atrophy can achieve what none of the currently available therapies can do to treat this rare disease. After positive data from a ...

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an intriguing case study might demonstrate a simple new treatment, with a child ...

Maximum mouth opening, which is lower in children with spinal muscular atrophy compared to healthy kids, can be accurately measured during the first 2 years of life. Here’s why that matters.