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 ...

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 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 ...

What goat breed is best for you? From Nigerian Dwarf to Alpine to Nubian, here’s what to know about some of the best types of ...

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 ...

BOSTON, February 24, 2025--(BUSINESS WIRE)--PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of ...