The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to SAT-3247, an oral treatment candidate for Duchenne muscular dystrophy (DMD) that ultimately aims to slow the progression of the genetic condition. The FDA awards this status to investigational medicines that are designed to treat serious,…
News
The experimental gene therapy RGX-202 has been found to be well tolerated at a high dose, with biomarker data indicating it is working as designed to increase production of the microdystrophin protein in boys with Duchenne muscular dystrophy (DMD). That’s according to updated interim findings from the ongoing…
A low dose of PGN-ED051, PepGen’s investigational exon 51-skipping therapy, safely increased dystrophin protein levels in people with Duchenne muscular dystrophy (DMD), according to early Phase 2 clinical trial data. The therapy’s effects either were comparable to or greater than what has been observed in studies of…
Pfizer has discontinued development of fordadistrogene movaparvovec, its investigational gene therapy for Duchenne muscular dystrophy (DMD), after recent Phase 3 trial data indicated a failure to improve motor function in boys with the neuromuscular disease. Patients who have already received the gene therapy in the CIFFREO…
Duvyzat (givinostat), a newly approved treatment for Duchenne muscular dystrophy (DMD), is now available in the U.S., according to an announcement from ITF Therapeutics, which will be selling the therapy. “Following the FDA [Food and Drug Administration] approval of Duvyzat, our team has been focused on making…
Most children with Duchenne muscular dystrophy (DMD) start experiencing notable issues with arm function and breathing ability before they lose the ability to walk, a new study reports. The findings have major implications for clinical trials testing treatments that aim to boost arm and/or lung function in DMD. Traditionally,…
The U.S. Food and Drug Administration (FDA) has given the green light for Myogenica to start a clinical trial testing its stem cell therapy MyoPAXon in people with Duchenne muscular dystrophy (DMD). DMD, the most common type of muscular dystrophy, is caused by mutations in the…
Satellos Bioscience is gearing up to launch a Phase 1 clinical trial of its Duchenne muscular dystrophy (DMD) treatment SAT-3247. The company said it has submitted an application to authorities in Australia seeking permission to start the trial, which is expected to test the safety and pharmacological properties…
Treatment with the gene therapy BB-301 led to improved swallowing for the first person with oculopharyngeal muscular dystrophy (OPMD) to receive the treatment as part of an ongoing clinical trial, according to new interim data from BB-301’s developer Benitec Biopharma. “We are pleased to report continued positive…
One year of losmapimod taken twice a day improved or stabilized upper limb function and muscle strength in adults with facioscapulohumeral muscular dystrophy (FSHD), according to an open-label pilot study. Blood and muscle tests also showed the therapy candidate successfully engaged with its intended target and had a…
Recent Posts
- Sarepta plans to seek full FDA approval of 2 Duchenne treatments
- When it comes to rare disease advocacy, just keep showing up
- MDA 2026: This year’s MDA meeting provided a glimpse into the future
- I’m thankful for a big family that gives my DMD sons a full social life
- MDA 2026: Dyne advances DMD therapy z-rostudirsen toward approval