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...

Spanish study: Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for myotonic dystrophy type 1 www.sciencedirect.com/science/arti...

Univ. of Albany/VCU/Univ. of Florida study: Modified Polycyclic Compounds Rescue Mis-splicing in Myotonic Dystrophy Type 1 Disease Models pubs.acs.org/doi/10.1021/...

Watershed moment: for first time results of clinical trial testing drug safety and efficacy in myotonic dystrophy T1 patients published in the New England Journal of Medicine.

Univ. of Albany/VCU/Osaka Univ. study: Identification of enzymatically modified isoquercitrin as a therapeutic lead for myotonic dystrophy type 1 academic.oup.com/narmolmed/ar...

New study: Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies (Dystro-miR1) clinicaltrials.gov/study/NCT074...

Italian study: Minute-by minute gait variations during the 6-Minute walk test in subjects with myotonic dystrophy type 1 journals.sagepub.com/doi/10.1177/...

Italian study: Quantitative susceptibility mapping in myotonic dystrophy: clinical relevance of subcortical iron accumulation watermark02.silverchair.com/fcag017.pdf?...

UCSD/Université Laval study: MBNL2 dysfunction in outer radial glial cells is associated with disrupted corticogenesis in congenital myotonic dystrophy www.sciencedirect.com/science/arti...

Univ. of Ottawa review: Disruptions of Cell Signaling Pathways in Myotonic Dystrophy Type 1 (DM1) Skeletal Muscle, their Pathogenic Impact and Potential for Combinatorial Therapeutics www.jbc.org/article/S002...

Fascinating, US researchers discuss a version of myotonic dystrophy impacting Quarter horses (they found no trinucleotide repeat expansions were not found in the DMPK 3'UTR (CTG)n nor tetranucleotide repeat expansions (CCTG)n in intron 1 of CNBP) journals.plos.org/plosone/arti...

Finnish study: Nuclear MBL-1 modulates mitochondrial morphology through carnitine palmitoyltransferase in Caenorhabditis elegans with toxic trinucleotide repeats www.pnas.org/doi/10.1073/...

#$RNA Avidity adjusts endpoints of del-desiran P3 trial in myotonic dystrophy T1 patients. Now measured at 54 weeks (was 30 weeks). Adds 10 minute walk test as secondary outcome measure clinicaltrials.gov/study/NCT064...

Int'l study: Large-scale Proteomics Profiling of Peripheral Blood of DM1 patients identifies biomarkers for disease severity and functional capacity www.medrxiv.org/content/10.1...

Canadian study: Endogenous circadian rhythm sleep disorders through the lens of nonparametric variables of actigraphy: an exploratory study in myotonic dystrophy type 1 www.researchgate.net/publication/...

Italian/French study: Translational behavioral phenotypes in DMSXL mice for CNS manifestations of DM1 journals.sagepub.com/doi/10.1177/...

Dutch study: The Rasch-transformed gastrointestinal symptoms rating scale in myotonic dystrophy type 1 (RT-GSRS-DM1) www.sciencedirect.com/science/arti....

Multi-center study: A pothole-filling strategy for selective targeting of rCUG-repeats associated with myotonic dystrophy type 1 www.pnas.org/doi/10.1073/...

Belgian study: Quantification of Interactions between Small Molecules and RNA Probes Representative of Myotonic Dystrophy Type 1 Using Affinity Capillary Electrophoresis and UPLC-UV pubs.acs.org/doi/10.1021/...

Spanish study: Genetic anticipation and cardiac conduction abnormalities in myotonic dystrophy type 1: implications for early stratification from a multicenter registry www.sciencedirect.com/science/arti...

Italian review: Myotonic dystrophy and cancer risk: Insights, limitations, and the need for genetic certainty www.jns-journal.com/article/S002...

French study: Predictors of quality of life and social participation in myotonic dystrophy type 1 www.em-consulte.com/article/1784...

Mexican study: Fifteen Years of Myotonic Dystrophy Type 1 in Mexico: Clinical, Molecular, and Socioeconomic Insights from a National Reference Cohort www.mdpi.com/2073-4425/16...

Univ of Florida study: Quantitative Magnetic Resonance Imaging of the Forearm in Myotonic Dystrophy Type 1 www.mdpi.com/2379-139X/11...

Multi-institution study: Atrial arrhythmia ablation in myotonic muscular dystrophy patients: a single institution experience link.springer.com/article/10.1...