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

Scientists found a genetic link between autism and DM1, where repeat DNA sequences disrupt brain gene splicing. This sheds ...

The antimalarial drug mefloquine could help treat genetic diseases such as cystic fibrosis, Duchenne muscular dystrophy, as ...

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

Jordan McLinn has been an advocate since childhood to add add Duchenne muscular dystrophy to Indiana’s newborn screening ...

Mike Pence in 2015 as he signed Indiana’s Right To Try Law, which allows terminally ill patients to access experimental ...

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

Researchers suggest discovery of genetic link between ASD and DM1 opens up potential new therapeutic avenues for ASD.

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 ...

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

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

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, ...