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 ...

Columnist Robin Stemple knows that his physical challenges aren't his fault, but that doesn't make it any easier to avoid feeling ashamed.

People with BMD who received sevasemten in clinical trials maintained stable motor function over several years of follow-up, new data show.

New Phase 1b data support the safety and effectiveness of salanersen over one year in children with SMA who had the potential for improvement due to suboptimal clinical status with prior gene ...

US biotech major Biogen has presented additional results from the Phase Ib study of salanersen, an investigational novel ...

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.

Permanent; Fitzroy location; Challenging and rewarding work environment; About the Role An opportunity is available for a Grade 3 Physiotherapist to join the ...

March 11 () - 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 ...

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 ...

The strife-marked Duchenne muscular dystrophy (DMD) space drew forth another outspoken political figure in the shape of Sen. Ron Johnson (R-Wisc.), who said he was “enraged” by the U.S. FDA’s refusal ...

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 ...