Dutch study: Generation of isogenic rescue iPSC lines by targeted CTG-repeat excision for myotonic dystrophy type 1 www.sciencedirect.com/science/arti...

Northeastern/Rockefeller/Wash U study: Bottlebrush polymer conjugates for enhanced antisense oligonucleotide therapy in myotonic dystrophy type 1 academic.oup.com/nar/article/...

Explore MDF's Exercise Guide for people living with #MyotonicDystrophy—packed with practical tips, gentle habits, and encouragement to help you move with confidence every day. 💚 Learn more: myotonic.org/wp-content/u...

MDF/Third Plateau study: Exploring barriers to clinical trial readiness among the myotonic dystrophy community: a mixed-methods study link.springer.com/article/10.1...

Int'l study: Exploring the impact of myotonia on daily functioning in myotonic dystrophy: a patient-reported survey link.springer.com/article/10.1...

German review: Current biomarker development in myotonic dystrophies link.springer.com/article/10.1...

French study: Determinants of diaphragm ultrasound and its diagnostic performance for predicting respiratory status in myotonic dystrophy type 1. www.sciencedirect.com/science/arti...

Quebec review: Gynecological and Obstetrical Issues Experienced by Women Diagnosed with Myotonic Dystrophy Type 1: A Scoping Review journals.sagepub.com/doi/10.1177/....

Korean study: Neurofilament light chain reflects motor impairment in myotonic dystrophy type 1 www.frontiersin.org/journals/neu...

Int'l study: Optical mapping reveals a higher level of large-scale structural variants in a family with paternally transmitted myotonic dystrophy and independent Parkinson's disease pathsocjournals.onlinelibrary.wiley.com/doi/10.1002/...

$IONS/Quebec study: Antisense-Mediated Gene Therapy Targeting DMPK Restores Cardiac Ion Channel Function and Electrical Stability in Myotonic Dystrophy Type 1 www.sciencedirect.com/science/arti...

Dyne Therapeutics Announces Completion of Enrollment in Registrational Expansion Cohort of ACHIEVE Trial of Z-Basivarsen for Myotonic Dystrophy Type 1 (DM1) finance.yahoo.com/sectors/heal...

Emory/Univ. of Florida study: Muscleblind-like proteins dimerize by forming disulfide bonds to regulate alternative splicing and pathogenic RNA foci formation academic.oup.com/nar/article/...

Chinese study: A pilot study on the application of Oxford Nanopore PromethION sequencing with adaptive sampling for DMPK repeat expansion sizing in myotonic dystrophy type 1 www.sciencedirect.com/science/arti...

Spanish review: The myotonic dystrophy type 1 drug development pipeline: 2026 Edition www.sciencedirect.com/science/arti...

Poster presentations for International Myotonic Dystrophy Consortium-15 conference in Quebec now available here: event.fourwaves.com/idmc-15/abst...

PR: ARTHEx Biotech Delivers Multiple Presentations at IDMC-15 Highlighting Clinical Progress of ATX-01 and Mechanistic Insights for Myotonic Dystrophy Type 1 (DM1) and 2 (DM2) www.prnewswire.com/news-release...

Spanish study: Oleic Acid Levels in HSALR Mouse Model of Myotonic Dystrophy Type 1 www.mdpi.com/1422-0067/27...

Very interesting Turkish study: Evaluating the performance of ChatGPT in responding to myotonic dystrophy type 1 patient inquiries: a specialist physician-based study link.springer.com/article/10.1...

$TRDA/Univ. of Florida study: Enhanced antisense oligonucleotide delivery reveals that transcript turnover impacts apparent splicing rescue in myotonic dystrophy www.cell.com/molecular-th...

Dutch study: Generation of iPSC lines from myotonic dystrophy type 1 patients with varying CTG repeat lengths www.sciencedirect.com/science/arti...

Int'l study: Longitudinal Psychometric Properties of the Myotonic Dystrophy Health Index in a Large Multicenter Cohort of People Living With Myotonic Dystrophy Type 1 onlinelibrary.wiley.com/doi/10.1002/...

Spanish study: Sex Differences in Cardiac Involvement in Adults With Myotonic Dystrophy Type 1: A Multicenter Study www.sciencedirect.com/science/arti...

Myotonic dystrophy-related abstracts from Modalis/Affinia (#1492), Univ. of Washington (#2502) and Ascidian (#2471) at ASGCT conference download.asgct.org/2026ASGCTAbs...

Quebec study: Short-term course of the neuropsychological profile in myotonic dystrophy type 1: a 12-month longitudinal study link.springer.com/article/10.1...

Quebec / SUNY / NYU review: Therapeutic Strategies Targeting the Molecular Pathogenesis of Myotonic Dystrophy Type 1: Current Status and Future Directions www.researchgate.net/publication/...

Korean study: Targeted long-read sequencing for high-resolution repeat profiling in myotonic dystrophy type 1 www.nature.com/articles/s12...

Multi-Center study: Effect of exercise training on clinical and physiological variables in adults with myotonic dystrophy type 1: a systematic review and meta-analysis link.springer.com/article/10.1...

Dutch study: Multidimensional Measurements of Dysarthria in Myotonic Dystrophy Type 1 onlinelibrary.wiley.com/doi/10.1111/...

Stanford/Greenstone Biosciences study: Generation and characterization of two human induced pluripotent stem cell lines from myotonic dystrophy type 1 patients www.sciencedirect.com/science/arti...

Developing endpoints for the cardiac burden in myotonic dystrophy type 1: A workshop report journals.sagepub.com/doi/10.1177/...

VCU/Int'l study: Toward Trial Readiness in Congenital Myotonic Dystrophy: A Longitudinal Cohort Study of Predictors of Motor Function in Childhood www.neurology.org/doi/10.1212/...

Emory/Univ of Florida study: Muscleblind-like proteins dimerize by forming disulfide bonds to regulate alternative splicing and pathogenic RNA foci formation www.biorxiv.org/content/10.6...

Polish review: MBNL proteins in health, disease, and therapeutic applications academic.oup.com/nar/article/...

Baylor study: Progressive cardiac phenotypes and reduced reversibility from long-term CUGexp RNA expression in a DM1 mouse model insight.jci.org/articles/vie...

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

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

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

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 www.aan.com/msa/Public/E...

#$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-...

Univ of Rochester/Stratevi/$DYN study: Clinical and healthcare burden of myotonic dystrophy type 1 (DM1) in the United States: a claims-based study www.tandfonline.com/doi/full/10....

Italian study: Intraocular pressure and corneal biomechanics in patients affected by myotonic dystrophy type 1 link.springer.com/article/10.1...

Int'l study using END-DM1 natural history data: Prospective Study of Video Hand Opening Time as a Quantitative Measurement of Myotonia in Patients With Myotonic Dystrophy Type 1 www.neurology.org/doi/10.1212/...

Dutch study: Response to noninvasive home mechanical vEntilation in Myotonic Dystrophy type 1: The multicenter REMeDY study journals.sagepub.com/doi/10.1177/...

“Subsequent to that partnership, in a fairly shocking and certainly ironic twist of fate, my personal commitment to muscular dystrophy deepened, as two members of my immediate family have been diagnosed now with myotonic dystrophy, DM1,” Ingram said... www.fiercepharma.com/pharma/sarep...

Japanese study: Elevated Risk of Endometrial Cancer and Precursor Lesions in Patients With Myotonic Dystrophy: A Retrospective Study at a Single Institution in Japan obgyn.onlinelibrary.wiley.com/doi/10.1111/...

Univ. of Rochester study: Test-Retest Reliability of Remote Assessments in Patients With Myotonic Dystrophy Type 1 www.neurology.org/doi/10.1212/...

ARTHEx Biotech Announces Publication in The American Journal of Human Genetics Highlighting the Discovery and Preliminary Preclinical Profile of ATX-01 for Myotonic Dystrophy Type 1 (DM1) www.prnewswire.com/news-release...

Quebec study: Modeling Myotonic Dystrophy Type 1 with hiPSCs-Derived Cardiac Organoids Reveals Key Disease Mechanisms www.heartrhythmjournal.com/article/S154...

Bunch of abstracts on myotonic dystrophy to be presented at MDA Clinical and Scientific Conference in March. Search "myotonic" here: www.mdaconference.org/abstracts/20...