Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and is eventually fatal.

Jun 20, 2024 · ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene. For patients who are non-ambulatory and have a confirmed mutation in the DMD gene.

Duchenne muscular dystrophy, sometimes called DMD or Duchenne, is a rare genetic disease. Duchenne is caused by a change (called a mutation) in the DMD gene, which prevents the body from making dystrophin, a protein found in muscle cells. Dystrophin protects muscles from injury. If the body can’t make dystrophin, or can’t make enough of it ...

Jun 22, 2023 · Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene between exons 1 to 17 and exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction.

Learn about treatment approaches for Duchenne muscular dystrophy (DMD) and explore options that may be right for you. Exon-skipping therapy involves skipping over a certain exon (or section of a gene), depending on a person's genetic mutation.

Learn how Sarepta therapies may help your patients living with Duchenne muscular dystrophy (DMD). Our SareptAssist patient support program helps your patients explore eligibility, coverage, financial assistance options, and treatment logistics. Get your patients started in just 3 steps: Our team offers information and resources on:

Jun 22, 2023 · Sarepta's muscular dystrophy treatment is the first gene therapy approved under the program. The disease, which almost exclusively affects boys, destroys muscles.

Jun 20, 2024 · Last year, the drug – Elevidys, from the biotech company Sarepta Therapeutics – was approved to treat only children ages 4 and 5 with Duchenne muscular dystrophy, one of the most severe forms of...

6 days ago · New data on Sarepta's gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have introduced a concern about its safety.

Sarepta is currently researching investigational gene therapies that utilize the AAVrh74 vector. A vector is a component of gene therapy that helps deliver a gene therapy into a cell. The EXPLORE DMD study will test for antibodies associated only with this vector.