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, dm1myotonic.bsky.social
Joined May 2013
- Tweets 2,730
- Following 127
- Followers 791
- Likes 1,853
114 Photos and videos
Pinned Tweet
17 Dec 2022
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/…
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German review: Current biomarker development in myotonic dystrophies link.springer.com/article/10…
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Jun 12
French study: Determinants of diaphragm ultrasound and its diagnostic performance for predicting respiratory status in myotonic dystrophy type 1. sciencedirect.com/science/ar…
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Jun 11
Quebec review: Gynecological and Obstetrical Issues Experienced by Women Diagnosed with Myotonic Dystrophy Type 1: A Scoping Review journals.sagepub.com/doi/10.….
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Jun 11
Korean study: Neurofilament light chain reflects motor impairment in myotonic dystrophy type 1 frontiersin.org/journals/neu…
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DM1Research retweeted
Our expedition has directed us to this point: Advancing an oral genetic medicine that is a pioneering, first-in-class small molecule that targets disease-driving RNA. Learn about the progress we have made in our DM1 program: arrakistx.com/myotonic-dystr…
#GoingFurtherForDM1
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DM1Research retweeted
Dr. John W. Day recently received the Steinert Clinical and Basic Sciences Award from the Myotonic Dystrophy Foundation at the 2026 IDMC-15 International Myotonic Dystrophy Consortium Meeting in Saguenay, Québec. Congrats, Dr. Day! #myotonicDystrophy 💚@MyotonicStrong
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Jun 9
Int'l study: Optical mapping reveals a higher level of large-scale structural variants in a family with paternally transmitted myotonic dystrophy and independent Parkinson's disease pathsocjournals.onlinelibrar…
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Jun 8
Quebec / $IONS study:
Jun 7
Antisense-Mediated Gene Therapy Targeting DMPK Restores Cardiac Ion Channel Function and Electrical Stability in Myotonic Dystrophy Type 1
heartrhythmjournal.com/artic…
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Jun 3
Dyne Therapeutics Announces Completion of Enrollment in Registrational Expansion Cohort of ACHIEVE Trial of Z-Basivarsen for Myotonic Dystrophy Type 1 (DM1) finance.yahoo.com/sectors/he…
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Jun 3
Emory/Univ. of Florida study: Muscleblind-like proteins dimerize by forming disulfide bonds to regulate alternative splicing and pathogenic RNA foci formation academic.oup.com/nar/article…
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May 31
Chinese study: A pilot study on the application of Oxford Nanopore PromethION sequencing with adaptive sampling for DMPK repeat expansion sizing in myotonic dystrophy type 1 sciencedirect.com/science/ar…
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May 31
Spanish review: The myotonic dystrophy type 1 drug development pipeline: 2026 Edition sciencedirect.com/science/ar…
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May 27
Poster presentations for International Myotonic Dystrophy Consortium-15 conference in Quebec now available here: event.fourwaves.com/idmc-15/…
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May 27
PR: ARTHEx Biotech Delivers Multiple Presentations at IDMC-15 Highlighting Clinical Progress of ATX-01 and Mechanistic Insights for Myotonic Dystrophy Type 1 (DM1) and 2 (DM2) prnewswire.com/news-releases…
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May 27
Spanish study: Oleic Acid Levels in HSALR Mouse Model of Myotonic Dystrophy Type 1 mdpi.com/1422-0067/27/10/421…
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DM1Research retweeted
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
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May 26
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…
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DM1Research retweeted
May 22
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/articl…
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DM1Research retweeted
May 21
$PEPG - PepGen Announces Presentations at the 15th International Myotonic Dystrophy Consortium Highlighting Clinical Data from the PGNEDODM1 Program
News & Disclaimer
ift.tt/C0S5d2i
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May 21
$TRDA/Univ. of Florida study: Enhanced antisense oligonucleotide delivery reveals that transcript turnover impacts apparent splicing rescue in myotonic dystrophy cell.com/molecular-therapy-f…
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