Researchers from University College London (UCL) are aiming to develop potential treatments for congenital muscular dystrophy ...

Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).

In the full study, conducted in collaboration with the Castiglione Lab at Vanderbilt University and partially funded by the ...

Globally, more than 350 million people live with rare diseases, most of them caused by a misstep hidden within their genes.

New research has uncovered a genetic connection between autism spectrum disorder (ASD) and myotonic dystrophy type 1 (DM1), a rare neuromuscular disease.

Ndeye Lam visits the cemetery often, praying and gently touching the seashells laid out across her daughter’s gravesite.

Scientists from The Hospital for Sick Children (SickKids) and University of Las Vegas Nevada (UNLV) have uncovered a genetic link between autism spectrum disorder (ASD) and a rare genetic condition ...