Marisa Wexler, MS,  senior science writer—

Through a program called MDA Engage, the Muscular Dystrophy Association (MDA) is offering virtual educational events for people affected by muscular dystrophy and other neuromuscular diseases. The events are aimed at people living with these conditions, as well as their loved ones. MDA Engage offers three types…

Treatment with Translarna (ataluren) increased levels of the protein dystrophin in young boys with Duchenne muscular dystrophy (DMD) caused by nonsense mutations, new clinical trial data show. The findings were presented in a webcast by Stuart W. Peltz, PhD, the CEO of PTC Therapeutics, which…

Applications are now open for the seventh annual Strategies to Realize Innovation, Vision, and Empowerment (STRIVE) Awards program for Duchenne muscular dystrophy. The annual program, facilitated by PTC Therapeutics, was designed to help support initiatives that address unmet needs in the Duchenne community. Goals of STRIVE range from…

A potential exon-skipping therapy for Duchenne muscular dystrophy (DMD) called DS-5141 showed a good safety profile with repeat dosing in a small clinical trial, according to a press release. The Phase 1/2 clinical trial (NCT02667483) tested DS-5141 for a first time in Duchenne patients amenable to exon 45 skipping. No…

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,”…