Sarepta Therapeutics Inc. (NASDAQ:SRPT) released three-year follow-up results from its EMBARK study, showing that its gene ...

Sarepta Therapeutics, Inc. , the leader in precision genetic medicine for rare diseases, today announced positive topline three-year functional results from Part 1-treated patients in EMBARK (Study ...

Zeleciment basivarsen (z-basivarsen) demonstrated sustained functional improvement across multiple clinical measures in the ongoing ACHIEVE ...

With a tank of zebrafish, Queensland researcher Jean Giacomotto was able to test a never-before seen genetic variant ...

The U.S. Food and Drug Administration has awarded orphan drug status to Atossa Therapeutics' (Z)-endoxifen for Duchenne ...

An international team of scientists has designed a new type of therapy to target the mutant toxic RNA that causes myotonic ...

New York, Jan. 26, 2026 (GLOBE NEWSWIRE) -- In recognition of Rare Disease Day on February 28, the Muscular Dystrophy Association (MDA) is sharing powerful community stories throughout the month that ...

By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...

Researchers at Kumamoto University have demonstrated that iron supplementation can significantly alleviate muscle pathology ...

Researchers at Kumamoto University have demonstrated that iron supplementation can significantly alleviate muscle pathology and functional decline in a mouse model of facioscapulohumeral muscular ...

Researchers in Class of 1942 Professor of Chemistry Matthew D. Shoulders' lab have uncovered a sinister hidden mechanism that ...

In mantle cell lymphoma (MCL), an aggressive form of B-cell non-Hodgkin lymphoma, TP53 mutations are known to affect patients’ prognosis—but questions remain. What does the heterogeneity of TP53 ...