Duchenne Muscular Dystrophy

Repurposing existing therapies may help slow muscle weakness in patients with Duchenne muscular dystrophy, a new study suggests.

Developed by Sarepta Therapeutics, Elevidys is a one-time, intravenous treatment that uses adeno-associated virus (AAV) technology to deliver a gene designed to produce a shortened form of the dystrophin protein, known as Elevidys micro-dystrophin, directly to skeletal muscle.

Muscle weakness has the biggest impact on patients’ ability to walk, but other factors matter too, researchers found.

Capricor officials said the company was prepared for the meeting and does not expect it to delay the FDA’s decision on the therapy.

A new report suggests combining the antimalarial drug with aminoglycosides could enhance the latter’s efficacy, which could also have important safety implications.

A trial found magnetic resonance imaging correlated with physical function tests in patients with Duchenne muscular dystrophy.

A new meta-analysis finds corticosteroids have long-term benefits, and a separate study suggests benefits hold regardless of dosing schedule.

A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.

Doing more to incorporate caregiver burden into value assessments and cost-effectiveness research of Duchene muscular dystrophy treatments was the focus of a recent International Society for Pharmacoeconomics and Outcomes Research (ISPOR) webinar.

Participants in the FORWARD-53 trial had clinically significant improvements in time-to-rise, among other metrics.

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of this year.

Six takeaways from a recent review about the brain-related comorbidities of Duchenne muscular dystrophy

The company says it plans to file for accelerated approval later this year.

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is priced at $3.2 million per dose, produced $821 million in net product revenue for the company in 2024.

The company is evaluating whether the drug, which targets cardiac complications, might be used treat patients with other diseases, such as systematic sclerosis.

Duvyzat was approved in March 2024 and works to reduce the inflammation and loss of muscle experienced by patients with Duchenne muscular dystrophy.

A number of physicians experienced in treating DMD across the nation contributed to this article recently published in the Annals of the Child of Neurology Society.

Full approval was granted for the one-time gene therapy to ambulatory patients aged 4 years older. The FDA also granted accelerated approval to Elevidys for non-ambulatory patients.

New White Paper looks at the solutions and policy options of how to pay for high-cost gene therapies when there is still so much uncertainty about the outcomes and the durability of clinical benefits.