Myotonic dystrophy type 1 (DM1), a genetic disorder, shares overlapping features with chronic kidney disease, including ...

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.

Desert Metals Ltd. ( ($AU:DM1) ) has provided an announcement. Desert Metals Ltd. has released its interim financial report for the half year ...

Details of the presentation are as follows: Arrakis’s DM1 drug program represents a rationally designed small molecule genetic medicine directed at a pathogenic RNA to achieve reversal of ...

Thurman M. Wheeler, MD, of the Department of Neurology at Massachusetts General Hospital, is the senior author of a paper published in Nature Communications, "Analysis of human urinary extracellular ...

Sarepta Therapeutics hits 52-week lows despite strong earnings and growth. Read why investors should view SRPT as a high risk ...

After PepGen (PEPG) announced initial clinical data from the five and ten mg/kg dose cohorts in the ongoing FREEDOM-DM1 Phase 1 trial investigating PGN-EDODM1 in myotonic dystrophy type 1 ...

Mis-splicing is the underlying cause of DM1 pathology, and we believe the mean splicing correction observed at 28 days following a single dose of PGN-EDODM1 at 10 mg/kg in the FREEDOM clinical ...

PepGen (PEPG) announced initial clinical data from the five and ten mg/kg dose cohorts in the ongoing FREEDOM-DM1 Phase 1 trial investigating PGN-EDODM1 in myotonic dystrophy type 1. Mean splicing ...

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 ...

PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies ...