The U.S. Food and Drug Administration (FDA) has given rare pediatric disease status to AMO Pharma‘s investigational therapy AMO-02 (tideglusib), now entering clinical testing in children and adolescents with congenital myotonic dystrophy type 1 (CDM1), also known as Steinert disease. “This Rare Pediatric Disease designation highlights the significant…
Ultragenyx Pharmaceutical and Solid Biosciences announced a strategic collaboration to develop and commercialize new gene therapies for Duchenne muscular dystrophy (DMD). The collaboration will combine Solid’s gene therapy construct for DMD and Ultragenyx’s cell…
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…
Parent Project Muscular Dystrophy (PPMD) is seeking adults with Duchenne or Becker muscular dystrophy to serve on the 2021 PPMD Adult Advisory Committee (PAAC). The committee is meant to represent the teen and adult voice of people living with these conditions, and to help their…
Catabasis Pharmaceuticals’ investigational oral therapy edasalonexent failed to preserve muscle function and overall functionality in boys with Duchenne muscular dystrophy (DMD), top-line data from the PolarisDMD Phase 3 clinical trial show. “We are deeply saddened and disappointed by the results of our Phase 3 PolarisDMD trial,” Jill C.
Long-term corticosteroid treatment delays respiratory decline and heart disease in boys with Duchenne muscular dystrophy (DMD) regardless of daily or intermittent use, a real-world study reported. The study, “Cardiorespiratory Progression Over 5 Years and Role of Corticosteroids in Duchenne Muscular Dystrophy: A Single-Site…
A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…
The 11th annual Ryan’s Day fundraiser, organized by rapper Wheelz to raise awareness of Duchenne muscular dystrophy and help find a cure, will be live-streamed on Facebook on Oct. 24 at 1 p.m. (ET). Official Ryan’s Day 11 Poster…
The Muscular Dystrophy Association Telethon returns this month after a six-year absence, with actor Kevin Hart hosting the virtual event. The two-hour MDA Kevin Hart Kids Telethon, set for Oct. 24 at 8 p.m. (ET), will stream live on the…
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…
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