… the US Food and Drug Administration (FDA). PepGen is also … most prevalent muscular dystrophy in adults. The current treatment landscape includes … running a Phase III trial (NCT06411288) of del- … open-label extension (OLE) trial. GlobalData predicts sales of …

… and Drug Administration (FDA) to pull the drug from the … treatment for Duchenne muscular dystrophy (DMD). The patients who died after taking the drug … Institute. “The FDA withheld the drug because Dr. Vinay … ,” said Orient. New Drugs, New Framework The science …

… to locally produce a vital drug for treating Spinal Muscular Atrophy (SMA … and swallowing. Current treatment options include gene therapy, medication and physical therapy … in treatments for other rare genetic diseases, such as Duchenne Muscular Dystrophy (DMD …

… .S. Food and Drug Administration (FDA) pioneered this approach … medications, coverage after approval is essential to ensure treatment … ” with pharmaceutical companies for new drugs that cannot … therapy for Duchenne muscular dystrophy that received accelerated …

FDA and Capricor aligned on endpoints for HOPE-3 pivotal trial … received Orphan Drug Designation for the treatment of Duchenne Muscular Dystrophy (DMD … with potential to treat and prevent a … (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory …

… release of groundbreaking clinical trial results. The clinical … Phase 1 study, treating patients with myotonic dystrophy … discontinuing its Duchenne muscular dystrophy programs earlier this year … therapeutic response. The treatment was generally well-tolerated …

… Duchenne muscular dystrophy.  The FDA turned down Capricor’s application for the drug, deramiocel … conference call with analysts. Initial trial results are expected in the … win approval of the first treatment for heart-related complications of …

Neurology/psychiatric The FDA has awarded orphan drug designation to Cure Rare … treatment of limb-girdle muscular dystrophy type R9 (LGMD2i/R9), a congenital muscular dystrophy … /congenital Neurology/psychiatric FDA Orphan drug

… trial data to the U.S. Food and Drug … Administration to get its Deramiocel therapy approved in the treatment … of cardiomyopathy associated with Duchenne muscular dystrophy. The FDA stated … Topline data from the trial, which will serve as …

… the Food and Drug Administration next year … on patients’ lives.” Treatment was also associated with … a confirmatory trial. Company executives said the FDA said … muscular dystrophy gene therapy Elevidys and, reportedly, skeptical of another Duchenne treatment …