Researchers have identified a way of changing parts of DNA sequencing to be used as a treatment for genetic diseases. The CRISPR/Cas9 therapy (or clustered regularly interspaced short palindromic repeats) is a new method of genome editing which can be used in the treatment of Duchenne muscular dystrophy. UT Southwestern researcher…
Spending more time outdoors, engaging in daily activities and living in a community that accepts patients and their families all tend to improve the lives of boys with Duchenne muscular dystrophy (DMD), a European analysis finds. The analysis,“Predictors of Health-Related Quality of Life in boys with Duchenne muscular dystrophy from six European…
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…
Winter is a perilous time if you have a compromised immune system, but you can’t stay alone inside for four months. You have to get on with your daily life, and just hope you don’t succumb to other people’s bugs and illnesses. However, there are some foods that can help you avoid catching a cold…
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…
A family from Torrington, Connecticut is taking advantage of the FDA’s temporary approval of the muscular dystrophy drug Exondys 51 (or Eteplirson). In a report from Fox61.com, mom Mel Kelly had joined an MD movement that applied pressure on the FDA to approve the drug. Find out more…
As boys with Duchenne muscular dystrophy get older, their disease progresses and it’s likely that their emotional response to the disease will change over time. It’s perfectly natural for Duchenne MD patients to experience frustration, anger, self-pity and anxiety regarding their condition, but there are ways that you can help…
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…
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