News

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to delpacibart etedesiran (del-desiran, previously called AOC 1001), an investigational treatment for myotonic dystrophy type 1 (DM1) that’s about to enter Phase 3 clinical testing. The FDA gives the designation to experimental therapies that have the…

Dosing has been paused in a Phase 3 trial testing fordadistrogene movaparvovec in children with Duchenne muscular dystrophy (DMD) following the sudden death of a boy who had been previously treated with the gene therapy, its developer Pfizer announced in a letter to the community. Pfizer indicated…

AMO Pharma has announced it will conduct a Phase 3 trial of AMO-02 (tideglusib), its investigational oral therapy for adult-onset myotonic dystrophy type 1 (DM1). The decision follows a recent meeting with the U.S. Food and Drug Administration (FDA) to review data from the Phase 2/3…

A $250,000 award from the Parent Project Muscular Dystrophy (PPMD) will support the establishment of Baby Duchenne, a collaborative clinical research network for babies with Duchenne muscular dystrophy (DMD) diagnosed via newborn screening (NBS) programs in New York state. Under the direction of Bo Hoon Lee, MD, from…

Researchers have discovered a new, unrecognized type of muscular dystrophy that’s caused by inherited mutations in the SNUPN gene, a study reports. Most people who carry the mutations develop symptoms of muscle weakness before age 2, and the muscles of the upper arms and legs are mainly affected. “This…

Most people with myotonic dystrophy type 1 (DM1) develop heart problems, according to a Danish study that followed patients for about 10 years. “Life-long cardiac screening is crucial, and we recommended that repeated follow-up is performed,” its researchers wrote in “Natural history of cardiac involvement in myotonic dystrophy type 1 – Emphasis…

Glucocorticoid use can help maintain breathing ability and arm function in adults with Duchenne muscular dystrophy (DMD), a new study reports. Importantly, the study found that glucocorticoid treatment was shown to be helpful even when only considering the time after patients had lost the ability to walk — which…

Oral treatment with EDG-5506, now known as sevasemten, continued to stabilize muscle function after two years in men with Becker muscular dystrophy (BMD) taking part in a Phase 1 trial. That’s in contrast to the progressive deterioration of motor function seen during the natural course of BMD in the absence…

Treatment with an antioxidant supplement led to improvements in muscle strength and quality of life for people with facioscapulohumeral muscular dystrophy (FSHD) in a small clinical trial. The use of such supplements was particularly seen to improve the muscle quality of patients’ quadriceps — a group of muscles found…

Diagnostic testing of individuals in India suspected of having limb-girdle muscular dystrophy (LGMD) revealed two novel mutations in the calpain-3 gene, known as CAPN3, that were found to cause the common subtype R1, known as LGMDR1. These findings were detailed in a new study reporting the results of testing…