Duchenne Muscular Dystrophy

Business

Sarepta's Gene Therapy Shows Sustained Slowing of DMD Progression in Landmark Three-Year Data

New long-term results from the pivotal EMBARK trial indicate ELEVIDYS, Sarepta's gene therapy, continues to alter the disease trajectory for boys with Duchenne muscular dystrophy, with no new safety concerns emerging over three years.

Business

PTC Therapeutics Gains Analyst Favor: Barclays Upgrade Fuels Valuation Debate Amid Rare Disease Focus

Barclays' upgrade of PTC Therapeutics to Overweight has spotlighted the biotech firm's valuation, with shares showing strong momentum but questions lingering over its path to profitability and reliance on a narrow drug portfolio.

FDA Rejects Aquestive's Oral Anaphylaxis Treatment, Leaving Patients Awaiting Epinephrine Alternative

The U.S. Food and Drug Administration has issued a complete response letter for Aquestive Therapeutics' oral film drug designed to treat severe allergic reactions, the company announced Monday, delaying a potential new option for millions at risk.