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Last week, my girlfriend and I caught the 2020 filmed version of Lin-Manuel Miranda’s award-winning Broadway production “Hamilton.” The recording, which we watched on Disney+, featured the original cast performing at the Richard Rodgers Theatre in New York City. Exactly a month ago, we attended a live performance of the musical with SMA News Today…

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After nearly a year of treatment, Viltepso (viltolarsen) was well tolerated and tended to increase how fast boys with Duchenne muscular dystrophy (DMD) could stand from a lying position, but not significantly more than a placebo, according to a preliminary analysis of a Phase 3 study. The main goal of that study, RACER53 (NCT04060199), was…

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NS Pharma, Inc. has shared preliminary analysis results from RACER53, the global Phase 3 clinical trial of NS-065/NCNP-01 (viltolarsen). Viltolarsen is an antisense oligonucleotide drug intended to treat patients with Duchenne who are amenable to exon 53 skipping. The drug was approved by the FDA in 2020 under the brand name VILTEPSO® under the FDA…

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As we prepare to welcome families to the CureDuchenne FUTURES Annual Conference, scientific leaders, executives from nearly every pharmaceutical and biotech company focused on developing therapies for Duchenne and Becker muscular dystrophy, and regulatory leaders came together for a discussion on addressing the challenges and opportunities in bringing treatments to individuals who need them. Peter…

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My oldest son, Max, 18, graduated from high school last Sunday. Graduation is a milestone for all who achieve it, but since Max lives with Duchenne muscular dystrophy (DMD), it felt like an even greater accomplishment. I was bursting with emotion, including joy, relief, happiness, and fear. It was overwhelming in the best way, and…

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As a funder of hC Bioscience, we’re happy to have them announcing their tRNA-based approach for targeting nonsense mutations at our annual Futures conference. The post hC Bioscience Announces Program in Duchenne Muscular Dystrophy and Reports In Vivo Data From tRNA-based Protein Editing Platform appeared first on CureDuchenne.

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Duchenne muscular dystrophy (DMD) can make it harder to engage in social activities, and patients often experience grief and frustration over lost function. Family, however, can be an invaluable source of support to help overcome the challenges of the disease. That’s according to a study, “‘You Take This Day by Day, Come What May’: A…

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The following content is sponsored by Edgewise Therapeutics and does not reflect the views of Muscular Dystrophy News or BioNews, Inc. Becker muscular dystrophy (Becker) is a serious genetic disease that imposes significant physical, emotional, financial, and social challenges on affected individuals and their families. Symptoms of Becker, including muscle wasting and cardiopulmonary deficits, can…

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Dyne Therapeutics has shared new clinical data from the ongoing DELIVER trial of DYNE-251 in patients with Duchenne who are amenable to exon 51 skipping. DYNE-251 is an exon skipping product that combines a PMO to enable skipping of exon 51 with a fragment antibody (Fab) to increase targeted delivery of the product to skeletal…

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PPMD is pleased to share that the article, “Barriers to diverse clinical trial participation in Duchenne muscular dystrophy: Engaging Hispanic/Latina caregivers and health professionals,” has been published in the Orphanet Journal of Rare Diseases. This qualitative study aims to understand the barriers faced by Hispanic/Latino families specifically and underrepresented groups more generally to clinical trial…

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