Focused on myotonic dystrophy type 1. My use of twitter/X is not an endorsement of the ninny who owns it. Follow me on Bluesky, https://nitter.cf/t.co/HrlGRPWgJw
Joined May 2013
- Tweets2.8K
- Following131
- Followers804
- Likes1.9K
Pinned Tweet
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/…
"Vertex is on track to share interim results from the GALILEO study in the second half of 2026." news.vrtx.com/news-releases/…
Norwegian study: Muscle Strength and Outcome Measures for Upper Limb Function in Adults With Myotonic Dystrophy Type 1: A Cross-Sectional Study onlinelibrary.wiley.com/doi/…
DM1Research retweeted
Share your thoughts on #myotonicDystrophy clinical trials with a 5-minute survey! Your feedback will help MDF understand what information is most important to the DM community as trials continue to develop. myotonicdistribution.qualtri…
NY study: Sympathetic neurons’ contribution to arrhythmias in a hiPSC-based myotonic dystrophy type 1 microphysiological system onlinelibrary.wiley.com/doi/…
Presentation by IU's Dr. Gutman: Z-Basivarsen Shows Functional Improvements in One-Year Data for Myotonic Dystrophy Type 1 youtube.com/watch?v=IdQ0b7Ja…
Dutch study: Exploring the gut microbiome in children and young adults with myotonic dystrophy type 1 journals.sagepub.com/doi/10.…
ASHG Abstract from Quebec: Leveraging a founder population to identify genetic modifiers of myotonic dystrophy type 1 ashg.floq.live/event/ashg-20…
ASHG abstract from Quest Diagnostics: Co-occurrence of CLCN1 variants with myotonic dystrophy repeat expansions (DM1/DM2) ashg.floq.live/event/ashg-20…
$IONS/Quebec/NYU study:
Antisense-mediated gene therapy targeting DMPK restores cardiac ion channel function and electrical stability in myotonic dystrophy type 1 #OpenAccess
heartrhythmjournal.com/artic…
Italian study: The cognitive-behavioral missing link in myotonic dystrophy type 1: Rethinking social cognition assessment journals.sagepub.com/doi/10.…
PR: Sarepta Therapeutics Presents New Preclinical Data Demonstrating Robust Knockdown of Pathogenic DMPK RNA in the Nucleus with SRP-1003 in DM1 at 2026 Oligonucleotide Therapeutics Society Annual Meeting finance.yahoo.com/healthcare…
Physical therapy professor Michael Kiefer awarded NIH Career Development Award to advance pediatric muscle disease research news.vcu.edu/article/physica…
Bunch of abstracts on myotonic dystrophy presented at World Muscle Society Conference Sept. 29-Oct. 3. Can search for full text of abstracts at link below. Will post some of titles in comments. nmd-journal.com/action/doSea…
