LoQus23 Therapeutics Ltd (“LoQus23”), a private biotechnology company investigating small molecule drugs that could stop the pathogenic triplet expansion that is the cause and driver of Huntington’s ...

With its recent data drop for an oligonucleotide candidate, Dyne Therapeutics signals it may become a frontrunner in this ...

Data from DELIVER and ACHIEVE Clinicals Trial to be Presented - - Presentation on ACHIEVE Trial in DM1 and Company Symposium to Feature Data on the Use of Splicing Correction as a Prognostic Biomarker ...

Myotonic dystrophy type 1 (DM1) is the most common muscular dystrophy that affects multiple systems including the muscle and heart. The mutant CTG expansion at the 3′-UTR of the DMPK gene causes the ...

A previous needle electromyogram had confirmed the presence of myotonia, but a muscle biopsy had revealed no evidence of dystrophy. Investigations Neurological examination, electrophysiological ...

Avas will commercialize NaMuscla for the symptomatic treatment of myotonia in adults with non-dystrophic myotonic (NDM) disorders. NaMuscla is the first and only licensed product for this indication.

In particular, this season shows Messer continuing to be at odds with Ali when the 15-year-old, who lives with Titin Myotonic Muscular Dystrophy, tries to assert more independence without mobility ...

Leah Messer is opening up about navigating her daughter Ali's muscular dystrophy as the 15-year-old takes a big step in her life: high school. In an interview with TooFab ahead of the new season ...

2025 could be a breakout year for the Duchenne muscular dystrophy space. Although several therapies have entered the market in recent years, there is still considerable unmet need for the 15,000 ...

This article was republished with permission from WTOP’s news partners at Maryland Matters. Sign up for Maryland Matters’ free email subscription today. A fourth case of bird flu was ...

has granted Fast Track designation for DYNE-101 for the treatment of myotonic dystrophy type 1 (DM1). DYNE-101 is currently being evaluated in the ongoing Phase 1/2 ACHIEVE global clinical trial.

They help doctors identify diseases that affect the nerves and muscles as well as the extent of any damage. This test is used to diagnose myotonic muscular dystrophy, which causes muscles to stay ...