Obtained ex-US Regulatory Clearance for DT-818, a Potentially Best-in-Disease Treatment for Myotonic Dystrophy Type-1 (DM1) Trials of DT-216P2 in Friedreich Ataxia (FA) and DT-168 in Fuchs Endothelial ...
September 6, 2007 — A new study using a mouse model of myotonic dystrophy type 1 (DM1) has revealed an early step in the process by which a mutant DM gene leads to cardiac defects. Found in more than ...
Avidity Biosciences, Inc. RNA provided an update on its phase I/II study of AOC 1001 in adults with myotonic dystrophy type I (DM1). The company reported that it is engaged in ongoing discussions with ...
WALTHAM, Mass.--(BUSINESS WIRE)--Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, today announced that ...
CAG and CTG (CWG) trinucleotide repeat expansions cause various neurological diseases, which can be classified as polyglutamate (polyQ) disorders (such as Huntington disease (HD)) with abnormal CAG ...
Myotonic dystrophy type 1 (DM1) is a multisystemic genetic disorder characterized by progressive muscle weakness and wasting, as well as several non-muscular complications such as cardiac, ...
Scientists from The Hospital for Sick Children (SickKids) and the University of Las Vegas, Nevada (UNLV) have uncovered a genetic link between autism spectrum disorder (ASD) and a rare genetic ...
Myotonic dystrophy type 1 (DM1) is a multi-systemic genetic disorder affecting the skeletal muscle, heart, brain, and other organs and is marked by progressive muscular weakness, atrophy and myotonia.
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