@dm1myotonic.bsky.social
Relaying the latest developments in the search for effective treatments for myotonic dystrophy type 1.
Serbian study: Sex-related phenotypic differences in myotonic dystrophy types 1 and 2: a nationwide registry study from Serbia link.springer.com/article/10.1...
Sex-related phenotypic differences in myotonic dystrophy types 1 and 2: a nationwide registry study from Serbia - Journal of Neurology
Background Myotonic dystrophies (DM) are autosomal dominant, multisystem disorders with variable clinical expressions. DM1 and DM2 share overlapping features, such as myotonia and muscle weakness, but...
link.springer.com
October 7, 2026 at 2:34 PM
Serbian study: Sex-related phenotypic differences in myotonic dystrophy types 1 and 2: a nationwide registry study from Serbia link.springer.com/article/10.1...
Italian study: Investigation of the pentamidine efficacy against myotonic dystrophy type 1 exploiting polymeric- and lipid-based nanoparticles www.sciencedirect.com/science/arti...
www.sciencedirect.com
October 5, 2026 at 1:17 PM
Italian study: Investigation of the pentamidine efficacy against myotonic dystrophy type 1 exploiting polymeric- and lipid-based nanoparticles www.sciencedirect.com/science/arti...
Jordanian/U.S. review: A Reassuring ECG Is Not Enough in Myotonic Dystrophy Type 1: A Systematic Review and Meta-analysis www.sciencedirect.com/science/arti...
www.sciencedirect.com
October 2, 2026 at 1:09 PM
Jordanian/U.S. review: A Reassuring ECG Is Not Enough in Myotonic Dystrophy Type 1: A Systematic Review and Meta-analysis www.sciencedirect.com/science/arti...
Swiss study: Antibody deficiency in myotonic dystrophy type 1: A differential diagnosis below the radar rupress.org/jhi/article/...
September 30, 2026 at 8:17 PM
Swiss study: Antibody deficiency in myotonic dystrophy type 1: A differential diagnosis below the radar rupress.org/jhi/article/...
Brazilian study: Lower urinary tract dysfunction in adults with myotonic dystrophy type 1: a multimodal clinical assessment www.sciencedirect.com/science/arti...
www.sciencedirect.com
September 27, 2026 at 11:23 PM
Brazilian study: Lower urinary tract dysfunction in adults with myotonic dystrophy type 1: a multimodal clinical assessment www.sciencedirect.com/science/arti...
Spanish review: Cognitive decline over time in myotonic dystrophy type 1: a systematic review of longitudinal studies www.frontiersin.org/journals/neu...
Frontiers | Cognitive decline over time in myotonic dystrophy type 1: a systematic review of longitudinal studies
BackgroundMyotonic Dystrophy Type 1 (DM1) is a multisystem genetic disorder and the most common muscular dystrophy in adults. Central nervous system involvem...
www.frontiersin.org
September 25, 2026 at 11:24 AM
Spanish review: Cognitive decline over time in myotonic dystrophy type 1: a systematic review of longitudinal studies www.frontiersin.org/journals/neu...
Univ. of Florida study: A mouse model of myotonic dystrophy type 1 exhibits pain-like behavior and peripheral nociceptor hyperexcitability www.biorxiv.org/content/10.6...
www.biorxiv.org
September 24, 2026 at 5:34 PM
Univ. of Florida study: A mouse model of myotonic dystrophy type 1 exhibits pain-like behavior and peripheral nociceptor hyperexcitability www.biorxiv.org/content/10.6...
Dutch study: Annual assessment of ECG intervals in patients with myotonic dystrophy type 1 using a mobile 6‑lead ECG device: TeleCheck-DM1 www.sciencedirect.com/science/arti...
Annual assessment of ECG intervals in patients with myotonic dystrophy type 1 using a mobile 6‑lead ECG device: TeleCheck-DM1
Cardiac involvement occurs in approximately 80% of patients with myotonic dystrophy type 1 (DM1), and annual follow-up with a routine 12‑lead ECG (rEC…
www.sciencedirect.com
September 15, 2026 at 1:23 PM
Dutch study: Annual assessment of ECG intervals in patients with myotonic dystrophy type 1 using a mobile 6‑lead ECG device: TeleCheck-DM1 www.sciencedirect.com/science/arti...
UPenn study: Advanced Conduction Disease After Incident Heart Failure in Myotonic Dystrophy Type 1 onlinelibrary.wiley.com/doi/10.1111/...
Advanced Conduction Disease After Incident Heart Failure in Myotonic Dystrophy Type 1
Central Figure. Advanced Conduction Disease After Incident HF in Myotonic Dystrophy Type 1.
onlinelibrary.wiley.com
September 11, 2026 at 12:52 PM
UPenn study: Advanced Conduction Disease After Incident Heart Failure in Myotonic Dystrophy Type 1 onlinelibrary.wiley.com/doi/10.1111/...
#$DYN Dyne Therapeutics to Present Additional One-Year Clinical Data from Phase 1/2 ACHIEVE Trial of Z-Basivarsen (DYNE-101) for Myotonic Dystrophy Type 1 (DM1) at Upcoming Medical Meetings investors.dyne-tx.com/news-release...
Dyne Therapeutics to Present Additional One-Year Clinical Data from Phase 1/2 ACHIEVE Trial of Z-Basivarsen (DYNE-101) for Myotonic Dystrophy Type 1 (DM1) at Upcoming Medical Meetings | Dyne Therapeutics, Inc.
- Data from a pooled dose group of participants initially enrolled in the multiple ascending dose portion of the trial to be compared against a matched natural history cohort - - Dyne also intends to provide the mean baseline value for video hand opening time (vHOT) in the ACHIEVE registrational
investors.dyne-tx.com
September 8, 2026 at 2:39 PM
#$DYN Dyne Therapeutics to Present Additional One-Year Clinical Data from Phase 1/2 ACHIEVE Trial of Z-Basivarsen (DYNE-101) for Myotonic Dystrophy Type 1 (DM1) at Upcoming Medical Meetings investors.dyne-tx.com/news-release...
Novartis provides update on delpacibart etedesiran (del-desiran) Phase III HARBOR study for the treatment of myotonic dystrophy type 1 (DM1). Drug failed to meet primary endpoint measuring efficacy. www.novartis.com/news/media-r...
Novartis provides update on delpacibart etedesiran (del-desiran) Phase III HARBOR study for the treatment of myotonic dystrophy type 1 (DM1)
www.novartis.com
September 8, 2026 at 10:58 AM
Novartis provides update on delpacibart etedesiran (del-desiran) Phase III HARBOR study for the treatment of myotonic dystrophy type 1 (DM1). Drug failed to meet primary endpoint measuring efficacy. www.novartis.com/news/media-r...
Int'l study: MBNL depletion drives stem cell fusion and immature myonuclear states in myotonic dystrophy type 1 www.nature.com/articles/s41...
MBNL depletion drives stem cell fusion and immature myonuclear states in myotonic dystrophy type 1 - Nature Communications
This study shows that muscle stem cells fuse with existing muscle fibres in myotonic dystrophy type 1, producing centrally located nuclei with altered gene-expression states and reshaping diseased mus...
www.nature.com
September 4, 2026 at 4:07 PM
Int'l study: MBNL depletion drives stem cell fusion and immature myonuclear states in myotonic dystrophy type 1 www.nature.com/articles/s41...
Novartis/Avidity study of Del-desiran for the Treatment of DM1 now "completed" as of July 29 on US govt clinical trials website clinicaltrials.gov/study/NCT064...
ClinicalTrials.gov
clinicaltrials.gov
September 1, 2026 at 12:25 PM
Novartis/Avidity study of Del-desiran for the Treatment of DM1 now "completed" as of July 29 on US govt clinical trials website clinicaltrials.gov/study/NCT064...
Couple of DM1 abstracts at ECS Congress. 1st: Spanish review, Long-term evolution in Myotonic Dystrophy Type 1: Predictors of cardiac involvement esc365.escardio.org/esc-congress...
ESC - Online Congress Platform
esc365.escardio.org
August 31, 2026 at 12:45 AM
Couple of DM1 abstracts at ECS Congress. 1st: Spanish review, Long-term evolution in Myotonic Dystrophy Type 1: Predictors of cardiac involvement esc365.escardio.org/esc-congress...
Virginia Commonwealth/Indiana University review: Myotonic dystrophy type 1 - updates on mechanisms of multisystemic manifestations, genetically targeted therapeutics, and preparing for a postapproval world. www.lifescience.net/publications...
Myotonic dystrophy type 1 - updates on mechanisms of multisystemic manifestations, genetically targeted therapeutics, and preparing for a postapproval world.
www.lifescience.net
August 24, 2026 at 11:16 AM
Virginia Commonwealth/Indiana University review: Myotonic dystrophy type 1 - updates on mechanisms of multisystemic manifestations, genetically targeted therapeutics, and preparing for a postapproval world. www.lifescience.net/publications...
Italian study: An Ultrastructural and Proteomic Analysis in DM1 Young Adults' Myoblasts: Stressed RER and Mitochondrial Dysfunction Involvement www.lifescience.net/publications...
An Ultrastructural and Proteomic Analysis in DM1 Young Adults' Myoblasts: Stressed RER and Mitochondrial Dysfunction Involvement.
www.lifescience.net
August 17, 2026 at 1:12 PM
Italian study: An Ultrastructural and Proteomic Analysis in DM1 Young Adults' Myoblasts: Stressed RER and Mitochondrial Dysfunction Involvement www.lifescience.net/publications...
Belgian study: Gastrointestinal manifestations are common and highly burdensome in patients with adult-onset myotonic dystrophy type 1 www.sciencedirect.com/science/arti...
Gastrointestinal manifestations are common and highly burdensome in patients with adult-onset myotonic dystrophy type 1
Myotonic dystrophy type 1 (DM1) is a progressive, multisystemic disorder in which gastrointestinal (GI) involvement is prevalent but frequently undere…
www.sciencedirect.com
August 6, 2026 at 9:01 PM
Belgian study: Gastrointestinal manifestations are common and highly burdensome in patients with adult-onset myotonic dystrophy type 1 www.sciencedirect.com/science/arti...
PR: PepGen to Advance PGN-EDODM1 Into Highest Dose Cohort in Phase 2 FREEDOM2-DM1 Study Following DSMB Review www.businesswire.com/news/home/20...
www.businesswire.com
August 6, 2026 at 12:44 PM
PR: PepGen to Advance PGN-EDODM1 Into Highest Dose Cohort in Phase 2 FREEDOM2-DM1 Study Following DSMB Review www.businesswire.com/news/home/20...
Japanese study: Total energy expenditure assessed by doubly labeled water in patients with myotonic dystrophy type 1: Associations with body composition and functional status journals.sagepub.com/doi/10.1177/...
Sage Journals: Discover world-class research
Subscription and open access journals from Sage, the world's leading independent academic publisher.
journals.sagepub.com
August 6, 2026 at 12:28 PM
Japanese study: Total energy expenditure assessed by doubly labeled water in patients with myotonic dystrophy type 1: Associations with body composition and functional status journals.sagepub.com/doi/10.1177/...
Canadian/Chinese study: A bitter melon natural compound ameliorates the myotonic dystrophy type 1 skeletal muscle phenotype in a sex-specific manner www.sciencedirect.com/science/arti...
www.sciencedirect.com
August 4, 2026 at 1:30 PM
Canadian/Chinese study: A bitter melon natural compound ameliorates the myotonic dystrophy type 1 skeletal muscle phenotype in a sex-specific manner www.sciencedirect.com/science/arti...
Update from #$VRTX on its myotonic dystrophy program: news.vrtx.com/news-release...
August 3, 2026 at 9:33 PM
Update from #$VRTX on its myotonic dystrophy program: news.vrtx.com/news-release...
Dutch study: Energy expenditure and the accuracy of predictive equations in myotonic dystrophy type 1 journals.sagepub.com/doi/10.1177/...
Sage Journals: Discover world-class research
Subscription and open access journals from Sage, the world's leading independent academic publisher.
journals.sagepub.com
August 3, 2026 at 1:13 PM
Dutch study: Energy expenditure and the accuracy of predictive equations in myotonic dystrophy type 1 journals.sagepub.com/doi/10.1177/...
Belgian study: 3D Radiomic Texture Analysis of Quantitative Muscle MRI Enhances the Distinction Between Myotonic Dystrophy Type 1 and Charcot-Marie-Tooth Neuropathy Type 1A: A Proof-of-Concept Study onlinelibrary.wiley.com/doi/full/10....
<fc>3D</fc> Radiomic Texture Analysis of Quantitative Muscle <fc>MRI</fc> Enhances the Distinction Between Myotonic Dystrophy Type 1 and Charcot–Marie‐Tooth Neuropathy Type <fc>1A</fc>: A Proof‐of‐Con...
In this proof-of-concept study, 3D radiomic texture analysis of quantitative muscle MRI (proton density fat fraction (PDFF, %) maps) distinguished the myogenic disease DM1 from the neurogenic disease....
onlinelibrary.wiley.com
August 3, 2026 at 1:06 PM
Belgian study: 3D Radiomic Texture Analysis of Quantitative Muscle MRI Enhances the Distinction Between Myotonic Dystrophy Type 1 and Charcot-Marie-Tooth Neuropathy Type 1A: A Proof-of-Concept Study onlinelibrary.wiley.com/doi/full/10....
Korean study: Differential expression of microRNAs and other small RNAs in the serum of patients with myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy type 1 www.sciencedirect.com/science/arti...
www.sciencedirect.com
July 31, 2026 at 12:46 PM
Korean study: Differential expression of microRNAs and other small RNAs in the serum of patients with myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy type 1 www.sciencedirect.com/science/arti...
Belgian study: Natural History of Adult-Onset Myotonic Dystrophy Type 1
Longitudinal Changes in Radiologic, Clinical, and Patient-Reported Outcomes www.neurology.org/doi/10.1212/...
Longitudinal Changes in Radiologic, Clinical, and Patient-Reported Outcomes www.neurology.org/doi/10.1212/...
Natural History of Adult-Onset Myotonic Dystrophy Type 1 | Neurology
Background and ObjectivesAdult-onset myotonic dystrophy type 1 (DM1) is a progressive, multisystemic disorder,
characterized by distal muscle weakness and myotonia. As disease-modifying therapies
emer...
www.neurology.org
July 30, 2026 at 12:05 AM
Belgian study: Natural History of Adult-Onset Myotonic Dystrophy Type 1
Longitudinal Changes in Radiologic, Clinical, and Patient-Reported Outcomes www.neurology.org/doi/10.1212/...
Longitudinal Changes in Radiologic, Clinical, and Patient-Reported Outcomes www.neurology.org/doi/10.1212/...