The classification of spinal muscular atrophy (SMA) subtypes, the influence of SMN2 gene copies on disease severity, treatment effects on disease progression, age-related differences, long-term ...

Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

Access to timely treatment for spinal muscular atrophy (SMA) remains a challenge, with barriers including delays in diagnosis, frequent treatment needs, and insurance coverage gaps, which impact ...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new 5-year data confirming the sustained efficacy and safety profile ...

The first program I want to go over is the use of taldefgrobep alfa, which is being developed in the phase 3 RESILIENT study for the treatment of patients with spinal muscular atrophy. Before going ...

After up to 4.8 years of continuous treatment with SPINRAZA, 100 percent of children treated pre-symptomatically were alive, and none require permanent ventilation Patients continued to maintain and ...

Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic ...

First and only muscle-targeted SMA therapy to demonstrate statistically significant, clinically meaningful improvements on the gold-standard HFMSE scale (p=0.019) versus placebo, with consistent ...

Imagine a child born in a family with a rare disease, whose treatment would cost more than 17 crore for a single dose. The ...