The symptoms of myotonic dystrophy may be obvious from birth or they can develop later -- during the teenage or adult years. Like other forms of muscular dystrophy, myotonic dystrophy leads to ...

Harmony Biosciences' Wakix shows promise for significant revenue growth. Find out why HRMY stock’s profitability and pipeline ...

If approved, paltusotine would become the first and only once-daily oral treatment available for acromegaly in adults ... designed to treat people with myotonic dystrophy type 1 (DM1), Duchenne ...

The FDA has granted fast track designation to DYNE-101, a therapy for myotonic dystrophy type 1 (DM1) now in testing in a ...

DYNE-101 Efficacy Data Today, Dyne reported efficacy data from adult DM1 patients enrolled ... Agency for the treatment of DM1. About Myotonic Dystrophy Type 1 (DM1) DM1 is a rare, progressive ...

RNA)’s stock is being fueled by encouraging clinical development of its 3 rare neuromuscular programs — del-desiran for myotonic dystrophy type 1 (DM1), del-zota for Duchenne muscular ...

"Up to one in five adults over the age of 70 have some ... the doctor said that conditions such as myotonic dystrophy, can be responsible for weakening the eyelid muscles. Damage to the eyelid's ...

DYNE-101 eased myotonia and led to other functional improvements for people with myotonic dystrophy type 1 (DM1) in a ...

Symptoms of Muscular Dystrophy Myotonic Muscular Dystrophy Also known as Steinert's disease or MMD, this is the most common form of adult muscular dystrophy. It starts with muscle weakness in the ...

Background medical conditions (number): short bowel syndrome (one), congenital heart disease (two), sickle cell disease (one), epilepsy (three), cerebral palsy (six), myotonic dystrophy (four ... In ...