DM1Research

If you or a family member have myotonic dystrophy, the #1 thing you can do to help researchers develop an effective treatment is to enroll in the END-DM1 natural history study. Details here: clinicaltrials.gov/ct2/show/NCT03…

Dutch study: Commitment to Myogenic Differentiation Significantly Aggravates the RNA Phenotype in Myotonic Dystrophy Type 1 onlinelibrary.wiley.com/doi/10.1111/na…

A first-of-its-kind national honor for research at @unlv. UNLV geneticist Łukasz Sznajder has been named the first recipient of the Muscular Dystrophy Association’s Research Momentum Award. Read more: conta.cc/4cMP84G @UNLV_CoS @MDAorg

Dutch/Univ. of Glasgow review: Expanding repeats, expanding impact: Somatic instability in myotonic dystrophy type 1 journals.sagepub.com/doi/10.1177/22…

ARTHEx Biotech Granted FDA Fast Track Designation for ATX-01 for the Treatment of Myotonic Dystrophy Type 1 (DM1) prn.to/4up2k5U

Delpacibart Etedesiran Improves the Molecular Pathology of Myotonic Dystrophy Type 1 in the Phase 1/2 MARINA® Study dlvr.it/TRQlrF #genetherapy #celltherapy

Today at the Muscular Dystrophy Association Clinical & Scientific Conference, Domi Stickens, PhD, CSO, presents a poster on our RNA-targeted small-molecule therapeutics and first oral therapy in development for myotonic dystrophy type 1 (DM1). #MDAconference #ArrakeensInAction

A Medical Case Study with Sarepta's CEO Doug Ingram at CNBC Cures Summit youtube.com/watch?v=efmKnJ…

French review: Targeting Expanded CUG and CTG Repeats as a Therapeutic Approach for Myotonic Dystrophy Type 1 (DM1) …mistry-europe.onlinelibrary.wiley.com/doi/10.1002/cm…

Zeleciment Basivarsen Targets the Underlying Cause of DM1 to Enable Functional Improvement in the Phase I/II ACHIEVE Trial index.mirasmart.com/AAN2026/PDFfil…

Resting Blood Pressure Patterns in Adults With Myotonic Dystrophy Type One and Type Two aan.com/events/annual-…

A few myotonic dystrophy related abstracts at AAN 2026 Conference. A Prospective Longitudinal Observational Study in Myotonic Dystrophy Type One: From Clinical Outcomes to Trial Design aan.com/msa/Public/Eve…

Interesting. Using "the five times sit to stand (5xSTS) test" as primary endpoint. That's not even a secondary endpoint in the Avidity del-desiran P3 trial; its primary endpoint is hand opening time.

@UFMedicine researchers developed safer Cas13-based RNA therapies that limit unintended RNA damage while treating repeat expansion disorders like myotonic dystrophy. The approach improves safety and enables in vivo delivery. License this #GatorMade tech: ufinnovate.technologypublisher.com/techcase/MP250…

$PEPG "The U.S. Food and Drug Administration (FDA) has placed a partial clinical hold on the FREEDOM2-DM1 Phase 2 multiple ascending dose (MAD), randomized, placebo-controlled clinical trial of PGN-EDODM1 in patients with myotonic dystrophy type 1" finance.yahoo.com/news/pepgen-an…

The U.S. National Institutes of Health has delayed posting calls for new grant applications for so long that large academic research programs may not have their funding renewed until next year—assuming those notices are approved at all. scim.ag/3OZmLpX

Heard on the Street: Biotech investors are increasingly wary of the rare-disease space, spooked by a regulatory shift under the Trump administration on.wsj.com/4bj4rAJ

Clinical value of routine 24-h Holter monitoring in the follow-up of patients with myotonic dystrophy type 1 #reccardioclinics #Aheadofprint reccardioclinics.org//es-clinical-v…

"Mean annual total cost of care was $20,687 (SD = $122,082), primarily driven by inpatient and outpatient hospital visits. Cardiac and pulmonary complications were significant predictors of high-cost status."

Univ of Rochester/Stratevi/$DYN study: Clinical and healthcare burden of myotonic dystrophy type 1 (DM1) in the United States: a claims-based study tandfonline.com/doi/full/10.10…

Italian study: Intraocular pressure and corneal biomechanics in patients affected by myotonic dystrophy type 1 link.springer.com/article/10.100…