A lightweight robotic knee device improves strength, muscle growth, and standing ability in children with SMA, with benefits ...

There is no single test to diagnose motor neurone disease. So it is generally only through referral to a neurologist, then ...

Strong Buy on DM1 & DMD clinical progress, BLA timeline, and strong cash runway through 2028. Click for this DYN update.

Primary endpoint is rise from floor (RFF) velocity with multiple secondary endpoints to assess muscle and pulmonary function ...

More than 1 million people across the United States live with multiple sclerosis (MS), a disease that affects the brain, ...

A rare and fatal brain disorder with no available treatment or cure. One that attacks the nervous system, balance, and the ability to move.

Multiple system atrophy (MSA) is a rare and fatal brain disorder with no available treatment or cure, attacking the nervous ...

The recent approval of Regeneron’s Otarmeni underscores the maturation of gene therapies across a range of diseases. Here, ...

A recent analysis of long-term health metrics reveals that adults with lower handgrip strength face a moderately higher risk ...

The FDA has an opportunity to reset its relationship with rare disease families and help, not obstruct. The new commissioner ...

Dyno Therapeutics, Inc., a genetic technologies company applying artificial intelligence (AI) to solve the grand challenge of in vivo gene delivery, today announced the launch of two new ...

The Muscular Dystrophy Association (MDA) today announced a $100,000 investment by BridgeBio to strengthen multidisciplinary care for people living with limb-girdle muscular dystrophy (LGMD). Through ...