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The U.S. Food and Drug Administration (FDA) has granted orphan drug status to RAG-18, being developed as a potential treatment for both Duchenne muscular dystrophy (Â鶹Éç) and Becker muscular dystrophy (BMD). RAG-18 is a small activating RNA (saRNA) therapy from Ractigen Therapeutics that’s designed to counteract the shortage…

A little over a year ago, neurologist Sarah Wright administered Elevidys (delandistrogene moxeparvovec-rokl) to then 5-year-old Hiram Secrist, making him the first Duchenne muscular dystrophy (Â鶹Éç) patient to receive the gene therapy outside of a clinical trial. Elevidys became the first and only gene therapy available for Â鶹Éç…

The U.S. Food and Drug Administration (FDA) has given WVE-N531, an investigational therapy for Duchenne muscular dystrophy (Â鶹Éç) patients amenable to exon 53 skipping, a rare pediatric drug designation. This status aims to incentivize companies developing treatments for serious or life-threatening conditions that primarily affect children and are considered…

Avidity Biosciences‘ delpacibart zotadirsen, or del-zota for short — formerly called AOC 1044 — was found to safely increase levels of dystrophin production to 25% of normal in people with Duchenne muscular dystrophy (Â鶹Éç), according to new data from the EXPLORE44 trial. In the Phase 1/2 clinical…

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 (Â鶹Éç) 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,…

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 (Â鶹Éç). 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 (Â鶹Éç), 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 (Â鶹Éç), 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 (Â鶹Éç), 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 (Â鶹Éç) 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 Â鶹Éç. Traditionally,…