A CRISPR-Cas9-based gene editing therapy promoted the production of a smaller but functional version of the dystrophin protein in a new mixed mouse model that uses muscle cells derived from Duchenne muscular dystrophy (DMD) patients. The therapy is designed to delete a DMD gene region that is commonly mutated in people…
The muscles of people with Duchenne muscular dystrophy (DMD) show profound impairment, with changes in fatty molecules and energy metabolism, as compared with those of their healthy peers, a small French study revealed. While previous studies have reported such metabolic changes in DMD patients, this was the first…
Following its acquisition last year by Astellas Pharma, Audentes Therapeutics now has become Astellas Gene Therapies. Named a Gene Therapy Center of Excellence, it will work to advance the development of potentially life-changing gene therapies for several conditions, including muscular dystrophy (MD). “The field of gene therapy has…
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. Long-term treatment with the experimental steroid therapy vamorolone safely and effectively delays motor function decline in boys 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. Audentes Therapeutics’ experimental exon skipping gene therapy may safely and effectively increase dystrophin levels and stabilize muscle function…
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. Monthly administration of SRP-5051 — Sarepta Therapeutics’ investigational, potentially improved version of its Exondys 51 (eteplirsen) therapy…
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…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…
The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…
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