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.
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.
Needham initiates Buy on Solid Biosciences with $16 target, highlighting potential of SGT-003 gene therapy amid concerns over Sarepta’s Elevidys safety.
FDA adds black box warning to Sarepta's (SRPT) Elevidys, limiting use to walking DMD patients after two deaths from liver failure. Stock rose 9% as investors relieved FDA maintained approval for walking patients rather than pulling drug completely.
Sarepta Therapeutics stock plunges as confirmatory trial for DMD drugs misses primary endpoint, adding to company's recent setbacks including Elevidys safety concerns.
BMO upgraded Sarepta stock, seeing high rewards despite drug safety worries. Elevidys sales are spiking, likely beating expectations. Future projects will bring new investors.