DALLAS, Texas — Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced multiple encore presentations supporting its TSHA-102 program in clinical evaluation for Rett syndrome...

CAMBRIDGE, Mass. — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today presented new data at the 31st Annual Congress of the World Muscle Society (WMS) that provide evidence of clinically meaningful functional benefit of ELEVIDYS (delandistrogene moxeparvovec) in older ambulatory patients with Duchenne muscular...

SAN DIEGO, Calif. — Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced research advancing understanding of Prader-Willi syndrome and expanding clinical and real-world evidence for VYKAT® XR (diazoxide choline) extended-release tablets will be presented at the Foundation for Prader-Willi Research (FPWR) 2026 Annual Research Symposium & Family Conference, taking place Oct. 7-10 in Philadelphia....