Sarepta just shared first test results for two new medicines aimed at rare muscle diseases FSHD1 and DM1. Early data show the drugs reach high levels in muscle safely, with no serious side effects. Shares jumped.
Major analysts like Citi, Needham, and Bank of America maintain Sell ratings on Sarepta after recent updates, citing risks to key products like Elevidys, lower 2026 outlook, and regulatory concerns in Duchenne muscular dystrophy treatments.
Sarepta rises as HHS recommends newborn screening for Duchenne muscular dystrophy and metachromatic leukodystrophy, aiming for earlier diagnosis and treatment.
Dyne Therapeutics' experimental DMD treatment shows strong results in late-stage trial, potentially offering more effective alternative to current controversial therapies with 10x higher dystrophin levels.
Novartis acquires Avidity Biosciences for $12 billion, gaining access to breakthrough RNA therapeutics for neuromuscular diseases as the Swiss drugmaker prepares for upcoming patent expirations on key drugs.
Oppenheimer downgrades Dyne Therapeutics to Perform from Outperform, maintaining $13 price target. New Phase 1/2 data shows promise, but valuation concerns prompt caution.
