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…
Edasalonexent, a potential oral therapy for Duchenne muscular dystrophy (DMD), can prevent the development of heart disease and help to preserve bone health, according to data from two studies in mouse models of DMD. Catabasis Pharmaceuticals’ investigational treatment was also well-tolerated when given for up to…
Santhera Pharmaceuticals has decided to stop SIDEROS, its Phase 3 trial assessing the effectiveness of Puldysa (idebenone) at delaying respiratory decline in patients with Duchenne muscular dystrophy (DMD) on a stable glucocorticoid dose. An interim data analysis of the study (NCT02814019), which was fully enrolled, determined that…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to PF-06939926, Pfizer’s investigational gene therapy for Duchenne muscular dystrophy (DMD). This designation is given to treatments that show considerable potential in addressing serious conditions for which available treatments fall short. It is…
Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…
The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on the Phase 1/2 trial testing SGT-001, Solid Biosciences’ investigational gene therapy for people with Duchenne muscular dystrophy (DMD), the company announced. Updated safety and effectiveness data for all patients dosed to date,…
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