Science-Based Medicine

Hope, Evidence, and Hard Questions in Duchenne Muscular Dystrophy

I’ve been reflecting on the disappointing outcomes and the reaction to Sarepta Therapeutics’ recently announced clinical ...
SMA News Today

New era of SMA care focuses on whole-body, long-term health

SMA expert Brian Lin, PhD, shares his thoughts on aging with SMA, treatment, advocacy, and supporting patients across their ...
Managed Healthcare Executive

Sarepta adds boxed warning to Elevidys about risk of liver disease

Elevidys now carries a black box warning for acute liver injury and failure, limiting its use to ambulatory patients aged four and older. The FDA's actions follow fatal liver failure reports in ...

FDA tightens Duchenne gene therapy after deaths of two teens

The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy ...
Precision Medicine Online

CHMP Recommends High-Dose Regimen of Biogen's Spinraza for Spinal Muscular Atrophy

The recommendation is based on the Phase II/III DEVOTE study testing the higher dose in treatment-naïve patients and those already on the low dose.
Laconia Daily Sun

FDA Limits Duchenne Gene Therapy After Two Teens Die of Liver Failure

MONDAY, Nov. 17, 2025 (HealthDay News) — The U.S. Food and Drug Administration (FDA) is tightening restrictions on a gene ...
Parade on MSN

These Are the 3 Best Exercises for Building Muscle, According to Decades of Research

Here are the three best exercises for building muscle, according to research (and experts): Squats “mimic many of our ...