HHS Adds Rare Diseases to Newborn Tests, Sarepta Rises
Sarepta rises as HHS recommends newborn screening for Duchenne muscular dystrophy and metachromatic leukodystrophy, aiming for earlier diagnosis and treatment.
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Dyne Therapeutics Hits Milestone with Promising Duchenne Drug, Eyes 2026 Approval
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 Makes Bold $12 Billion Move with Avidity Biosciences Acquisition in Largest Deal Under CEO Narasimhan
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.
Despite Positive DYNE-101 Data, Oppenheimer Steps to the Sidelines on Dyne Therapeutics Due to Valuation and Safety Caution
Oppenheimer downgrades Dyne Therapeutics to Perform from Outperform, maintaining $13 price target. New Phase 1/2 data shows promise, but valuation concerns prompt caution.