Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. The investigational gene therapy SGT-001 continues to lead to improvements in physical functioning, disease-related biomarkers, and patient-reported outcomes…
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Factors related to age, medication use, and co-existing conditions predict the likelihood of a person with…
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. A potential gene therapy known as SRP-9003 led to sustained production of beta-sarcoglycan in muscle…
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Gene therapy redosing after therapeutic plasma exchange (TPE) — done to lower antibodies against…
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. A long diagnostic process and a wide range of symptoms and functional problems affecting quality of life…
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. During the COVID-19 pandemic, people with certain muscular dystrophies reported moderate stress and challenges in…
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Switching from prednisone to Emflaza (deflazacort) successfully met efficacy and tolerability goals in most patients with Duchenne…
Next week’s 2021 MDA Virtual Clinical and Scientific Conference is expected to attract more than 1,200 clinicians and researchers from across the U.S. with expertise in neuromuscular conditions. Hosted by the Muscular Dystrophy Association (MDA), the conference runs March 15 to…
ReveraGen BioPharma has been awarded a $3.3 million grant from the National Institute of Neurological Disorders and Stroke (NINDS) at the National Institutes of Health (NIH) to support further development of vamorolone…
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…
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