Myotonic dystrophy can also impact the heart's electrical ... It has been described as congenital onset, juvenile onset, and adult onset, based on the age at which the symptoms begin.

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

People with myotonic dystrophy type 1 (DM1), the most common adult-onset form of muscular dystrophy, progressively lose ...

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 ...

“There are more than 100,000 adults with DM1 in the US and Europe ... ARTHEx also has ATX-01 in preclinical testing for myotonic dystrophy type 2 (DM2). “Although DM2 is caused by a different ...

The FDA has granted fast-track designation to an investigational antisense oligonucleotide to treat individuals with myotonic ...

We also investigate the pathogenic mechanisms of myotonic dystrophy, type 1 (DM1), an autosomal dominant ... in which failure to express adult splicing patterns causes primary features of the disease.

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

(RTTNews) - Dyne Therapeutics, Inc. (DYN), Tuesday announced that the U.S. Food and Drug Administration has granted Fast Track designation for DYNE-101 for the treatment of myotonic dystrophy type 1, ...

About Myotonic Dystrophy Type 1 (DM1) DM1 is a rare, progressive, genetic disease that affects skeletal, cardiac and smooth muscle. It is a monogenic, autosomal dominant disease caused by an abnormal ...

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 ...