Myotonic dystrophy type 1 is a rare, dominantly inherited, progressive, disabling, neuromuscular disease that leads to decreased life expectancy and has no approved therapies. The disease is caused by ...

ARTHEx Biotech, a clinical-stage biotechnology company advancing RNA-based therapeutics for neuromuscular disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast ...

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 the most common adult-onset form of muscular dystrophy and a condition that severely affects multiple organs including skeletal muscle, heart, brain and the ...

Its lead program, ATX-01, is in clinical evaluation for myotonic dystrophy type 1 (DM1), a rare neuromuscular disorder, in the Phase I/IIa ArthemiR™ trial. Building on this foundation, ARTHEx is ...

A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1 -- the most prevalent form of adult-onset muscular dystrophy -- provides insight into why patients develop fatty liver ...

HARMONIA trial will assess multi-system efficacy, safety and tolerability of z-basivarsen in DM1 -- 48-week trial will enroll approximately 150 ...

Scientists at 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 condition ...

Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy, affecting about 1 in 8,000 people. While it is well known for causing muscle weakness and stiffness, DM1 also ...

Myotonic dystrophy is a condition that causes thinned muscles, decreased muscle tone, and muscle weakness. Over time, a person may lose their strength and have issues relaxing their muscles. The ...

Delpacibart etedesiran led mostly to mild to moderate adverse events in a phase I/II trial of myotonic dystrophy type 1, but two serious events occurred. The antibody-oligonucleotide conjugate reduced ...