Marisa Wexler, MS, senior science writer —

AMO Pharma has announced the initiation of REACH-CDM, a pivotal Phase 2/3 clinical trial that will assess the efficacy and safety of the investigational medication AMO-02 (tideglusib) in children and adolescents with congenital myotonic dystrophy type 1 (CDM1), also known as Steinert disease. The double-blind trial (NCT03692312) is…

Translarna (ataluren) has been approved in Russia for treating Duchenne muscular dystrophy (DMD) caused by nonsense mutations in patients ages 2 and older. “Translarna was the first therapeutic ever approved for nonsense mutation Duchenne muscular dystrophy patients and we are excited to expand approval of Translarna into Russia,” Stuart…

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is central to NORD’s mission and history — community…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

In a Facebook Live event hosted by the Muscular Dystrophy Association (MDA), experts discussed the effect that the COVID-19 pandemic has had on research efforts in muscular dystrophy, as well as the ongoing development of a potential COVID-19 vaccine. Clinical trials are a critical…

Real-world and natural history data could be used to supplement, or perhaps replace, the use of a placebo in future clinical trials in people with Duchenne muscular dystrophy (DMD), a new study suggests. The study, “Suitability of external controls for drug evaluation in Duchenne muscular dystrophy,”…

An imbalance in cells producing proteins called aldehyde dehydrogenases (ALDHs) — favoring fat formation in tissue — is evident in the muscles of people with Duchenne muscular dystrophy (DMD), a study reports. A better understanding of these cells could open new avenues of treatment, its researchers said. The…

An application requesting the approval of casimersen (SRP-4045) to treat Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping has been accepted and placed under priority review by the U.S. Food and Drug Administration (FDA), Sarepta Therapeutics announced. A regulatory decision is expected no later than Feb. 25, 2021. The…

The LELANTOS Phase 3 clinical trial, which is evaluating FibroGen‘s pamrevlumab in people with Duchenne muscular dystrophy (DMD) who are no longer able to walk, has enrolled a first patient. This global study is currently recruiting up to 90 boys and men, ages 12 and older, using…

The importance of communication and self-care for people with muscular dystrophy and other neuromuscular diseases who are pursuing their education during the COVID-19 pandemic was highlighted in a Facebook Live event hosted by the Muscular Dystrophy Association (MDA). The event featured…