Patricia Inácio, PhD, science writer —

Muscular Dystrophy News Today brought you daily coverage of the latest scientific findings, treatment developments, and clinical trials related to muscular dystrophy (MD) throughout 2021. We look forward to reporting more news to patients, family members, and caregivers dealing with MD during 2022. Here are the top 10 most-read articles of…

A branch of the U.S. Food and Drug Administration (FDA) known as the Division of Cardiology and Nephrology has given positive feedback to Stealth BioTherapeutics‘ program for Duchenne muscular dystrophy (DMD). In a pre-investigational new drug (pre-IND) meeting, the division — called the DCN — agreed to the design of…

A Phase 1 trial of SRP-9001, Sarepta Therapeutics‘ investigational gene therapy, is recruiting 3-year-old boys with Duchenne muscular dystrophy (DMD) who are able to walk for a new patient group in California, according to a press release from the Muscular Dystrophy Association. This group, being enrolled at the…

Tamoxifen, commonly used to treat breast cancer, failed to meet its primary goal — delaying disease progression — in a Phase 3 study evaluating its use in boys with Duchenne muscular dystrophy (DMD), preliminary data show. Boys treated with tamoxifen and those given a placebo over 48 weeks in the…

CureDuchenne has teamed up with Brigham and Women’s Hospital, in Boston, to launch the nation’s first free newborn screening initiative for Duchenne muscular dystrophy (DMD). The partnership establishes Brigham and Women’s as the only U.S. birth hospital to offer parents the choice of screening their new baby for…

A treatment combination of two blood pressure medicines can help slow heart function decline in males with Duchenne muscular dystrophy (DMD), a single center study suggests. These findings support international guidelines that recommend the use of cardiac medications to help stabilize heart function in DMD. The study, “…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000 in grants, totaling…

For the first time, a new mouse model has been shown to mimic the heart disease features — specifically, to progress into late-stage heart failure, similar to humans — of people with Duchenne muscular dystrophy (DMD), a study reports. Moreover, using the new mouse model, researchers gathered promising data…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…