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

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 ...

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

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 trial ...

Avidity Biosciences specializes in RNA-based therapies using Antibody-oligonucleotide conjugates to deliver drugs directly to muscle and heart cells. Learn more on RNA stock here.

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

PepGen (PEPG) announced initial data from the FREEDOM-DM1 trial highlighting a dose- dependent robust increase in the splicing correction at 28 ...

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