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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. Treatment with Amondys 45 was safe and well-tolerated, showing little to no evidence of accumulation in the blood…

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…

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…

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…

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights? These are the colors most often used to represent Rare Disease Day. This year,…

The final patient has finished the first, six-month portion of the pivotal VISION-DMD Phase 2b clinical trial, assessing the anti-inflammatory steroid therapy vamorolone in boys with Duchenne muscular dystrophy (DMD). Top-line data from this part of…

The first patient has been dosed in a Phase 2 trial testing BBP-418, a potential first oral disease-modifying therapy for limb-girdle muscular dystrophy type 2i (LGMD2i). BBP-418 is a small molecule that delivers a prodrug — a precursor molecule — which, once inside the body, will change into the sugar…

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…

With the recent approval of Amondys 45 (casimersen) by Sarepta Therapeutics, there are now four exon-skipping therapies available in the U.S. for eligible patients with Duchenne muscular dystrophy (DMD). The others are Exondys 51 (eteplirsen) and Vyondys 53 (golodirsen), also by Sarepta, and Nippon Shinyaku’s…

The U.S. Food and Drug Administration (FDA) has conditionally approved Amondys 45 — formerly known as casimersen — by Sarepta Therapeutics as the first treatment for people with Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping. Accelerated, or conditional, approval is granted to a medication whose immediate availability fulfills an unmet medical…