Givinostat shows promise in delaying Duchenne muscular dystrophy (DMD) progression, enhancing mobility, and maintaining safety in long-term treatment.

DYNE-251 Granted FDA Breakthrough Therapy Designation for DMD

By Rose McNulty

August 13th 2025

DYNE-251 received FDA breakthrough therapy designation, showcasing its potential for patients with Duchenne muscular dystrophy (DMD).

Following an investigation, the FDA confirmed that shipments of delandistrogene moxeparvovec can resume. | Image Credit: immimagery - stock.adobe.com

FDA Recommends Sarepta Resume DMD Gene Therapy Shipments for Ambulatory Patients

By Rose McNulty

July 31st 2025

Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a voluntary pause.

The authors reported robust correlations between composite mean greyscale values and key functional outcomes in DMD. | Image Credit: hafakot - stock.adobe.com

Quantitative Muscle Ultrasound a Promising Noninvasive Biomarker for DMD

By Kimberly Rath, PharmD

July 29th 2025

Quantitative muscle ultrasound correlates strongly with ambulatory and timed function tests in Duchenne muscular dystrophy, suggesting it could complement or even replace more burdensome assessments.

The primary outcome measure in the ambulatory cohort in the study was mean change from baseline to week 52 in the TTR from floor, and no significant differences were seen between the treatment and control groups. | Image credit: OlegKachura - stock.adobe.com

TAS-205 Misses Primary End Point in Phase 3 DMD Trial

By Rose McNulty

July 11th 2025

TAS-205 showed no significant impact on motor function in patients with Duchenne muscular dystrophy (DMD), highlighting the ongoing search for effective treatments for the rare condition.

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