Margarida Maia, PhD, science writer —

The European Medicines Agency (EMA) has started reviewing an application to market givinostat for Duchenne muscular dystrophy (DMD). “Now that our market authorization application is under review, we are looking forward to working with the regulatory authorities to bring this treatment option to patients,” Paolo Bettica, MD, PhD,…

Pamrevlumab, an antibody in FibroGen’s pipeline, did not improve motor skills in boys with Duchenne muscular dystrophy (DMD), ages 6 to 11, who could walk, according to top-line data from a Phase 3 study. The LELANTOS-2 (NCT04632940) trial tested against a placebo pamrevlumab’s safety and how…

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease status to SAT-3153, Satellos Bioscience’s first-in-class therapy candidate to rebuild muscle tissue in Duchenne muscular dystrophy (DMD). “Receiving orphan drug designation and rare pediatric disease designations are important milestones in advancing our…

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy status to NS-089/NCNP-02, NS Pharma’s candidate for Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping, the company announced. The designation is intended to speed up the development and review of therapies for serious or life-threatening…

Mental health medications, including the stimulant methylphenidate, may help ease neurobehavioral problems in boys and young men with Duchenne muscular dystrophy (DMD), according to a small study by researchers in Belgium and the Netherlands. However, some adverse reactions…

Arrowhead Therapeutics has asked for permission to launch a clinical trial of ARO-DUX4, an experimental RNA-based medicine for facioscapulohumeral muscular dystrophy (FSHD) type 1, in New Zealand. The company’s application, filed with the New Zealand Medicines and Medical Devices Safety Authority, will be reviewed by the Standing…

In the U.S., only a limited number of patients will enter Sarepta Therapeutics‘ ENVISION, a Phase 3 study that’s part of the company’s strategy to gain global approval of SRP-9001 (delandistrogene moxeparvovec) as a gene therapy for Duchenne muscular dystrophy (DMD). These DMD patients will be non-ambulatory, or…

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on an application to launch a Phase 1 study of PGN-EDODM1, an experimental medicine by PepGen for people with myotonic dystrophy type 1 (DM1). A clinical hold is an order issued by the FDA to delay…

The U.S. Food and Drug Administration (FDA) has pushed back its decision on SRP-9001 (delandistrogene moxeparvovec), an experimental gene therapy for Duchenne muscular dystrophy (DMD). A decision is now expected by June 22, according to a press release. If the agency decides to approve SRP-9001, it’s expected to be…

IPS Heart has received rare pediatric drug designation for its GIVI-MPC and ISX9-CPC, two experimental single-course stem cell therapies for Duchenne muscular dystrophy (DMD). The designation is given by the U.S. Food and Drug Administration (FDA) to spur treatments for any rare disease that can be serious or…