She visited Fann hospital in Dakar, where neurologist Dr. Pedro Rodriguez measured her lung capacity. He suspects Mariama had ...

People with diseases caused by rare mutations have fewer options and poorer prospects than other patients despite rapid ...

These names in the biotech sector are seeing a substantial increase in search activity today, as determined by InvestingChannel. They include: ...

The orphan drug designation is particularly noteworthy as it can provide Dyne with a range of benefits, including reduced regulatory fees, assistance with clinical protocols, eligibility for research ...

Recently presented data demonstrated sustained functional improvement with DYNE-251 treatment through 18 months - - Data from the fully enrolled DELIVER registrational expansion cohort is planned for ...

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

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

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

John Brandsema, MD, a pediatric neurologist at Children’s Hospital of Philadelphia, speaks with Healio about the current understanding of the genetic factors behind muscular dystrophy.

John Brandsema, MD, a pediatric neurologist at Children’s Hospital of Philadelphia, speaks with Healio about the latest ...

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

A recent discovery of a molecular connection between autism and myotonic dystrophy, a type of neuromuscular disease, may provide a breakthrough on how clinicians approach autism spectrum disorder.