Diagnosis Recessive myotonia congenita (Becker's disease). Management Explanation of the nature of the disease and treatment with mexiletine 200 mg twice daily.

A one-year-old girl will fly to Mexico to receive potentially life-changing stem cell therapy after her family raised ...

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

“Evidence of treatment-induced elevation of MBNL in ... ARTHEx also has ATX-01 in preclinical testing for myotonic dystrophy type 2 (DM2). “Although DM2 is caused by a different mutation ...

Dyne Therapeutics (NASDAQ:DYN) reported clinical data from its ongoing Phase 1/2 trial of DYNE-101 in patients with myotonic dystrophy type 1. Shares down about 22% in early trade. DYNE-101 continued ...

Dyne Therapeutics Inc. is eyeing accelerated approval for its myotonic dystrophy type 1 treatment after reviewing new results from a phase I/II study. DYNE-101, an oligonucleotide antisense and DMPK ...

Request a sample and discover the recent advances in myotonic dystrophy treatment drugs @ Myotonic Dystrophy Pipeline Report The myotonic dystrophy pipeline report provides detailed profiles of ...

The PD-1 receptor, a major immune checkpoint inhibitor whose signaling is the target of multiple blockbuster anticancer drugs, differs functionally between rodents and humans in previously unknown ...