Author Archives: Marta Figueiredo PhD

A team of researchers received a $3-million grant from the Chan Zuckerberg Initiative to create a comprehensive blueprint of healthy skeletal muscle cells — whose dysfunction is associated with several rare muscle diseases, such as muscle dystrophy — as children grow. Muscular dystrophy comprises a group of more…

The U.S. Social Security Administration (SSA) has added Duchenne muscular dystrophy (DMD) to its Compassionate Allowances (CAL) program list, accelerating the disabilities application process for adults with this genetic disorder. The program is designed to identify severe medical diseases and conditions that automatically meet the SSA’s standards for…

A Phase 1b clinical trial evaluating Astellas Pharma’s ASP0367 is recruiting boys with Duchenne muscular dystrophy (DMD), ages 8–16, at six U.S. sites. The ongoing trial (NCT04184882) is evaluating the safety and tolerability of the investigational oral therapy, which is designed to improve muscle function in patients with DMD,…

Long-term treatment with Viltepso (viltolarsen) safely and effectively prevents a decline in motor function in boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Those are the findings of two-year data from a Phase 2 trial of Viltepso, an exon-skipping therapy being developed by…

CureDuchenne has launched the first comprehensive data hub to connect biosamples and clinical and genetic data from people with Duchenne and Becker muscular dystrophies — as well as disease carriers — with clinicians, researchers, and therapy developers. Called CureDuchenne Link, this next-generation data-sharing platform is expected…

High levels of the toxic RNA molecule associated with myotonic dystrophy type 1 (DM1) lead to defects in muscle regeneration and in the number of muscle stem cells — or satellite cells — and promotes muscle fibrosis (scarring) upon injury, according to a study in a mouse model of DM1.

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…