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.

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 ...

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 ...

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 ...