Targeting Expanded CUG and CTG Repeats as a Therapeutic Approach for Myotonic Dystrophy Type 1 (DM1) DM1 is an RNA gain-of-function disease caused by CTG repeat expansion, producing toxic r(CUG)exp RNA that sequesters MBNL1 and impairs splicing. This review covers the field of CUG and CTG ligands id...

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/...

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

Resting Blood Pressure Patterns in Adults With Myotonic Dystrophy Type One and Type Two www.aan.com/events/annua...

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...

PepGen Announces Regulatory Updates on FREEDOM2 BOSTON, March 04, 2026--PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies to treat severe neuromuscular and neurological dis...

$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-...

Clinical and healthcare burden of myotonic dystrophy type 1 (DM1) in the United States: a claims-based study Myotonic dystrophy type 1 (DM1) is a multi-systemic disease affecting skeletal and smooth muscle, the eye, brain, and heart. No disease-modifying therapies are available. Prior studies on clinical ...

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....

Intraocular pressure and corneal biomechanics in patients affected by myotonic dystrophy type 1 - Graefe's Archive for Clinical and Experimental Ophthalmology Graefe's Archive for Clinical and Experimental Ophthalmology - To compare corneal biomechanical properties in patients affected by Myotonic Dystrophy (DM1) with healthy subjects in order to...

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

"It seems that vHOT is not suitable to assess disease progression, but stability over 1 year may support its use to assess improvement."

Prospective Study of Video Hand Opening Time as a Quantitative Measurement of Myotonia in Patients With Myotonic Dystrophy Type 1 | Neurology Background and ObjectivesDelayed grip relaxation is a common symptom of myotonic dystrophy type 1 (DM1), differing from other muscle diseases. Preclinical studies suggest myotonia may reverse quickly ...

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/...

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

Sarepta CEO Doug Ingram to retire after a decade of Duchenne breakthroughs and controversies Sarepta Therapeutics CEO Doug Ingram announced Wednesday that he will retire by the end of 2026, citing a “shocking and ironic twist of fate” involving the health of his family. | Sarepta Therapeutics...

“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...

OBGYN Aim Myotonic dystrophy (MD) has been associated with an increased risk of endometrial cancer (EC) in Western countries; however, data from Japan are limited. This study aimed to evaluate the inciden...

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/...

Test-Retest Reliability of Remote Assessments in Patients With Myotonic Dystrophy Type 1 | Neurology Background and ObjectivesWe previously demonstrated the feasibility of remote assessments in individuals with myotonic dystrophy type 1 (DM1). This study aimed to evaluate test-retest reliability and ...

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) /PRNewswire/ -- Arthex Biotech, a clinical-stage biotechnology company advancing RNA-based therapeutics for neuromuscular disorders, today announced the...

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...

Modeling Myotonic Dystrophy Type 1 with hiPSCs-Derived Cardiac Organoids Reveals Key Disease Mechanisms Myotonic dystrophy type 1 (DM1) is a multisystemic disorder characterized by conduction defects and arrhythmias that contribute significantly to cardiac morbidity and sudden death. Human induced pluri...

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

2026 Abstract Library

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...

Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for myotonic dystrophy type 1 Myotonic dystrophy type 1 (DM1) is a multisystemic disorder caused by CTG repeat expansions in DM1 protein kinase (DMPK). Mutant transcripts containin…

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...

Modified Polycyclic Compounds Rescue Mis-splicing in Myotonic Dystrophy Type 1 Disease Models Myotonic dystrophy type 1 (DM1) is an autosomal dominant multisystemic disorder with no approved therapeutics targeting the disease mechanism. DM1 is caused by the expression of expanded CUG repeat RN...

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...

Minute-by minute gait variations during the 6-Minute walk test in subjects with myotonic dystrophy type 1 - Fabio Alexander Storm, Eleonora Diella, Luca Emanuele Molteni, Morena Delle Fave, Giovanni M... Background: Myotonic Dystrophy Type 1 is a rare multisystem disorder, with symptoms including progressive muscle weakness, myotonia and fatigue. Although mobili...

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?...

MBNL2 dysfunction in outer radial glial cells is associated with disrupted corticogenesis in congenital myotonic dystrophy Myotonic dystrophy type 1 (DM1) arises from toxic CUG-expanded DMPK transcripts that sequester Muscleblind-like (MBNL) proteins, yet how this molecula…

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...

Disruptions of Cell Signaling Pathways in Myotonic Dystrophy Type 1 (DM1) Skeletal Muscle, their Pathogenic Impact and Potential for Combinatorial Therapeutics Myotonic Dystrophy type 1 (DM1) is caused by a CUG expansion located in the 3’ untranslated region (UTR) of dystrophia myotonica protein kinase (DMPK) mRNAs. The pathogenic model underlying DM1 implic...

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...

Aberrant skeletal muscle morphogenesis and myofiber differentiation characterize equine myotonic dystrophy Equine myotonic dystrophy (eMD) is a rare neuromuscular disorder of undetermined origin marked by muscle hypertrophy and stiffness, dystrophic muscle histopathology, and myotonic discharges. In humans...

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...

Nuclear MBL-1 modulates mitochondrial morphology through carnitine palmitoyltransferase in Caenorhabditis elegans with toxic trinucleotide repeats | PNAS Expansion of nucleotide repeat sequences is linked to a growing number of neuromuscular degenerative disorders. Metabolic changes, including disrup...

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...

Large-scale Proteomics Profiling of Peripheral Blood of DM1 patients identifies biomarkers for disease severity and functional capacity Background Myotonic Dystrophy Type 1 (DM1), the most common genetic neuromuscular disorder in adults, poses significant challenges for drug development due to its multisystem nature and high clinical ...

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...

Endogenous circadian rhythm sleep disorders through the lens of nonparametric variables of actigraphy: an exploratory study in myotonic dystrophy type 1 | Request PDF Request PDF | On Jan 1, 2026, Luc Laberge and others published Endogenous circadian rhythm sleep disorders through the lens of nonparametric variables of actigraphy: an exploratory study in myotonic d...

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/...