News

The current incentives offered by the U.S. Food and Drug Administration (FDA) are not efficiently stimulating orphan drug development for rare conditions like muscular dystrophy and may be benefitting pharmaceutical companies more than patients, suggest a study published in the Orphanet Journal of Rare Diseases. This is evidenced by the unmet needs of patients with such diseases and the high price of newly approved orphan drugs.

Through in-depth interviews, adult patients with Duchenne muscular dystrophy (DMD) reveal their experiences and difficulties from the point of diagnosis, through disease progression, and how their attitudes changed over time. Transitioning to a wheelchair or a respirator were identified as triggers of anxiety and worry, and Duchenne patients at…

Apabetalone (RVX-208) may become a promising therapy to treat facioscapulohumeral muscular dystrophy (FSHD), according to results announced by the pharmaceutical company Resverlogix. Researchers found this drug acts by switching off the DUX4 gene, which is abnormally active in the muscles of FSHD patients, causing inflammation and muscle atrophy. Apabetalone…

The commercialization of the recently approved drug EMFLAZA™ (deflazacort) for the treatment of Duchenne muscular dystrophy (DMD) has been paused to reconsider the pricing of the drug.

Just days after Emflaza (deflazacort) became the first corticosteroid approved in the U.S. to treat Duchenne muscular dystrophy (DMD) regardless of mutation, the drug’s developer, Marathon Pharmaceuticals, announced it is “temporarily pausing” its sale in the U.S. to address concerns among the DMD community, particularly about the therapy’s high list price…

The strength of neck-flexing muscles not only affects torso movements but also the overall daily performance of children with Duchenne muscular dystrophy (DMD), a study has found. A key implication is that taking steps to maintain neck muscle strength early in the disease may improve the children’s movements. The research also…

The U.S. Food and Drug Administration (FDA) yesterday announced the approval of the corticosteroid drug deflazacort (under the trade name Emflaza) for the treatment of Duchenne muscular dystrophy (DMD).

An update on the current therapeutic approaches being developed to treat Duchenne muscular dystrophy (DMD) has been published in the journal Pharmacotherapy.

Researchers suggest a mutation in the INPP5K gene caused a new type of congenital muscular dystrophy. Individuals affected by the unique mutation also have a short stature, intellectual disabilities, and cataracts. The study, “Mutations in INPP5K Cause a Form of Congenital Muscular Dystrophy Overlapping Marinesco-Sjögren Syndrome and Dystroglycanopathy,” was a…

Two non-invasive strategies for measuring muscle function and its properties, electromechanical delay and ultrasound shear wave elastography, may help detect muscle impairments and assist in monitoring Duchenne muscular dystrophy (DMD) patients’ responses to therapy. The study “Effects of Duchenne muscular dystrophy on muscle stiffness and response to electrically-induced muscle…