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…

At the International Myotonic Dystrophy Consortium this week, Domi Stickens, PhD, Chief Scientific Officer, presents a poster on our RNA-targeted small-molecule therapeutics and the first oral therapy in development for myotonic dystrophy type 1 #DM1. #IDMC15 #MyotonicDystrophy

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/10.118…

I almost hesitate to promote this, because it wasn't really intended to be a piece. I just sort of sat down and it came out. Maybe someone else out there has the same type of day today, and it'll speak to them. realclearpolitics.com/articles/2026/…

$PEPG - PepGen Announces Presentations at the 15th International Myotonic Dystrophy Consortium Highlighting Clinical Data from the PGNEDODM1 Program News & Disclaimer ift.tt/C0S5d2i

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

A chemoinformatics-guided platform for efficient discovery of RNA-binding small molecules: Proof-of-concept for myotonic dystrophy type 1 | bioRxiv biorxiv.org/content/10.648…

🆕 At the end of work week we invite you to check out our recent article "Quantitative Magnetic Resonance Imaging of the Forearm in Myotonic Dystrophy Type 1" 🔹brnw.ch/21x2wz6 #magneticresonanceimaging #fatfraction #upperlimb #myotonicdystrophytype1 #myotonia #MRI

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

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/mu…

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

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

Fiscone et al. used quantitative susceptibility mapping to measure iron in cortical and subcortical regions of patients with myotonic dystrophy type 1. They observed increased iron in the thalamus and brainstem 👉 buff.ly/gAAF2Ul #braincommunications

There are many sterile stories about the farm crisis, and what tariffs and war have done to exacerbate it. If you read one story on the subject, it should be this one, about the final days of a multi-generational family farm. It'll rip your heart out. nytimes.com/2026/05/03/us/… via @NYTimes

Quebec study: Short-term course of the neuropsychological profile in myotonic dystrophy type 1: a 12-month longitudinal study link.springer.com/article/10.118…

📣MDF announces the launch of #myotonicDystophy grants aimed at accelerating efforts toward effective treatments & a cure. 🧬🔬Open to for-profit and non-profit institutions around the world.🌐💚🔗Share to your network & apply now: myotonic.org/myotonic-dystr…

Quebec / SUNY / NYU review: Therapeutic Strategies Targeting the Molecular Pathogenesis of Myotonic Dystrophy Type 1: Current Status and Future Directions researchgate.net/publication/40…

Korean study: Targeted long-read sequencing for high-resolution repeat profiling in myotonic dystrophy type 1 nature.com/articles/s1227…

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/10.100…

Dutch study: Multidimensional Measurements of Dysarthria in Myotonic Dystrophy Type 1 onlinelibrary.wiley.com/doi/10.1111/14…

A decade ago, a new class of Duchenne drugs sparked a civil war in the FDA. There were anecdotes, boys and parents in dire need, but very little data Today, those drugs are back. And for a small subset of patients, they have produced some of the best data experts have ever seen