Regenxbio has guided its Duchenne muscular dystrophy (DMD) gene therapy candidate through another test, reporting a clean ...

Turning his personal challenges into innovation, a wheelchair-bound mechanical engineering graduate from Veermata Jijabai Institute of Technology (VJTI) in Mumbai has developed a compact robotic ...

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

STELLAR-2, a randomized, double-blind, sham-controlled study, will evaluate the effects of salanersen when initiated ~6 months after onasemnogene abeparvovec-xioi in infants with SMA who received ...

Regenxbio Inc. RGNX shares are down on Wednesday after the company reported new interim data from its Phase 1/2 AFFINITY DUCHENNE trial of RGX-202. • Regenxbio stock is among today’s weakest ...

Solid Biosciences (SLDB) stock dips after new Phase 1/2 trial data for SGT-003, its gene therapy for Duchenne muscular dystrophy. Read more here, ...

Clinical & Scientific Conference, researchers and clinicians are talking about the central role of patients, families, and caregivers in driving change for people living with neuromuscular diseases.

Regenxbio said ​on Wednesday ‌that an ​interim ​data from a ⁠early-to-mid ​stage ​study of its experimental ​gene ​therapy in patients with ‌Duchenne ⁠muscular dystrophy showed continued ​improvement ...

Capricor Therapeutics’ deramiocel was rejected in July 2025, potentially caught between Nicole Verdun, a former top biologics regulator at the FDA, and Vinay Prasad, director of the Center for ...

Cheri Gunvalson spoke in Washington on Tuesday, seeking FDA flexibility for ataluren, a drug vital to her son Jacob, who has Duchenne muscular dystrophy.

Dyne Therapeutics is plotting an approval application for z-rostudirsen in the back half of 2026—a push that will only be bolstered by the departure of controversial CBER chief Vinay Prasad, according ...

The resubmission follows the positive results from the HOPE-3 Phase 3 trial. ・The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of Aug. 22, 2026. ・The investigational therapy ...