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 ...

Reduced progression of myocardial fibrosis measured by LGE on cardiac MRI (p=0.022)Significant improvement in LVEF versus placebo in patients ...

Detailed price information for Benitec Biopharma Ltd ADR (BNTC-Q) from The Globe and Mail including charting and trades.

This story is free to read because readers choose to support LAist. If you find value in independent local reporting, make a donation to power our newsroom today. In an eagerly anticipated decision, ...

The Muscular Dystrophy Association (MDA) concluded its 2026 MDA Clinical & Scientific Conference yesterday, convening over 2,400 attendees from 40 countries to help shape the future of neuromuscular ...

Consensus directed medical therapy (CDMT) was used in 73.1% of DMD patients with moderate or severe left ventricular dysfunction, leaving nearly 30% without recommended treatment. Target doses were ...

WASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting more than 25%. The young ...

Four patients with LGMD-R5 (gamma-sarcoglycanopathy, formerly LGMD-2C) have received the ATA-200 gene therapy as part of the ongoing Phase 1b/2 trial evaluating the safety, pharmacodynamics, and ...

In an eagerly anticipated decision, the Food and Drug Administration Thursday approved the first gene therapy for muscular dystrophy. "Today's approval addresses an urgent unmet medical need and is an ...