On March 3, 2025, in Ashburn, Virginia, USA, @0rionthelion shared a video of their cat with myotonia congenita. In the video, the cat is seen making a silly face with its tongue out. This genetic ...

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

Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, today announced that the company will present ...

Dyne Therapeutics’ DYNE-101 shows positive results in phase 1/2 ACHIEVE study. Read why DYN stock may rise with FDA approval and a $2.78B market opportunity.

Arrakis’s DM1 drug program represents a rationally designed small molecule genetic medicine directed at a pathogenic RNA to achieve reversal of myotonia in an animal model of DM1. Utilizing the ...

AOC 1001 improved myotonia (muscle stiffness) as measured by the vHOT test, and also hand grip strength and overall muscle strength using the QMT composite score. ADL, or Activities of Daily ...

In the Phase I/II ACHIEVE trial, key findings included DMPK RNA level reductions, CASI-22 improvements, early and sustained improvement in myotonia as measured by grip myotonia assessment, and ...

Correspondence to Dr Emma Matthews, MRC Centre for Neuromuscular Diseases, UCL and National Hospital for Neurology and Neurosurgery, London WC1N 3BG, UK; emma.matthews{at}ucl.ac.uk In case series we ...

Myopathy refers to diseases of skeletal muscles (muscles connected to bone). The condition can be acute (meaning sudden, severe, and short-lasting) or chronic (persistent, recurrent, and often ...