News

Throughout 2023, Muscular Dystrophy News Today worked diligently to cover new scientific research, treatment developments, and clinical studies for muscular dystrophy (MD). Here are the 10 most-read stories of that year. No. 10 – With EMBARK trial data, Sarepta seeks to expand Elevidys’ approval Elevidys (delandistrogene moxeparvovec-roki) is a…

People with Becker muscular dystrophy (BMD) caused by specific mutations tend to be younger when they first start using a wheelchair, a new study reports. The study, “Natural history of Becker muscular dystrophy: a multicenter study of 225 patients,” also provides new insights into the frequency of…

Disease progression with Becker muscular dystrophy (BMD) varies considerably, and depends on a patient’s age — with progression largely seen only in adults — and type of mutation, a three-year study of BMD’s natural history shows. “We … provide evidence that decline/improvement in function would be hard to measure…

HuidaGene Therapeutics’ investigational gene-editing therapy, called HG302, for Duchenne muscular dystrophy (DMD) has been granted a rare pediatric drug designation by the U.S. Food and Drug Administration (FDA). This status is intended to incentivize companies to develop treatments for rare and serious or life-threatening diseases affecting people under…

The European Commission has approved Agamree (vamorolone), a dissociative corticosteroid — a treatment expected to have fewer side effects than standard steroid medications — for patients ages 4 and older with Duchenne muscular dystrophy (DMD). The decision applies to all member states of the European Union, plus Iceland,…

In October, the U.S. Food and Drug Administration (FDA) approved Agamree (vamorolone) for treating Duchenne muscular dystrophy (DMD) patients ages 2 and older, offering them a more tolerable, but similarly efficacious, oral corticosteroid option. Clinical trial evidence indicates Agamree might ease muscle damage and minimize the side effects…

The HOPE-3 clinical trial testing Capricor Therapeutics’ experimental cell therapy CAP-1002 for Duchenne muscular dystrophy (DMD) can continue as planned, the company announced. That’s the result of an interim futility analysis, which evaluates whether it is mathematically possible for a trial to achieve its goal — if not, the trial…

Corticosteroids may cause fewer unwanted brain changes in boys with Duchenne muscular dystrophy (DMD) if they are given on and off at 10-day intervals, rather than at daily doses, an imaging study found. “Brain volumetrics [volume measurements] are more profoundly affected in patients treated with a daily corticosteroid…

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to Edgewise Therapeutics‘ EDG-5506 as a potential treatment for Duchenne muscular dystrophy (DMD). EDG-5506 also was awarded FDA orphan drug status for Becker muscular dystrophy (BMD) as a possible…

Growth hormone (GH) or testosterone can better protect against spinal fractures than a common osteoporosis medication alone in boys with Duchenne muscular dystrophy (DMD) or severe Becker muscular dystrophy (BMD), according to a long-term study. Used to prevent or lessen osteoporosis, or weak and brittle bones, zoledronic acid (ZA) is…