Abnormal values of immunologic parameters in myotonic dystrophy. Results of a study of relatives in 3 generations of a family dlvr.it/T6XnWH

AMO Pharma finishes meeting with US FDA and reveals plans for further clinical development of AMO-02 (tideglusib) for Myotonic Dystrophy treatment.

More Info: pryzm.ozmosi.com/product/2200 $XBI $IBB $XPH $PPH

Sex linked recessive - Color blindness, haemophilia

Autosomal recessive - sickle cell anemia , phenylketonuria, thalassemia

Autosomal dominant - myotonic dystrophy

Chromosomal - Downs, kleinfelters, turners

If you’re attending the annual ISPOR meeting, be sure to catch our poster presentation, “Costs and Healthcare Resource Utilization Evaluation in Myotonic Dystrophy Type 1: Results from the Real-world CARE-DM1 Study.' Register here: bit.ly/4b2V7hX #ISPORAnnual

Meet John Capecci and Timothy Cage from Living Proof Advocacy, along with Give an Hour’s Director of Programs, Julie Wells, co-presenting at the Myotonic Dystrophy Foundation’s Chicago Support Group Facilitator Training.

To Learn More, visit GiveAnHour.Org

French study: Heterogeneity of cognitive impairments in myotonic dystrophy type 1 explained by three distinct cognitive profiles pubmed.ncbi.nlm.nih.gov/38709306/

Don't miss it! ArrowheadPharma ($ARWR) is kicking off its summer series of R&D webinars on Thurs. May 23 at 2pm ET. Part I will feature Lawrence Korngut, MD who will discuss ARO-DM1 in type 1 myotonic dystrophy and ARO-DUX4 in FSHD. Register: brnw.ch/21wJmo8

Exciting News! 🎉
We're thrilled to announce the award an MSCA Doctoral Network to multiple European labs working on DM1.
We'll be recruiting talented PhD students next year.
Stay tuned for details! 🔍

#MSCA #myotonic Centre of Research in Myology (UMRS 974) Inserm Myotonic Dystrophy Foundation Institut de Myologie

We were honored to welcome members from the Myotonic Dystrophy Foundation & individuals from the myotonic dystrophy community to our facility. Our #Dynamos delivered presentations and provided a tour, fostering meaningful connections. A heartfelt thank you to everyone who joined us.

Learn more about one of MDF's 2024 Research Grant Recipients, Dr. Tahereh Kamali! Based in Dr. John Day's lab at the University of Stanford, Dr. Kamali is #researching the effects of #MyotonicDystrophy on the central nervous system. Learn more at: myotonic.org/early-career-r…

‏💥M ARSHID Ch💥 Figen These are Myotonic or 'Fainting Goats'. It's a disorder that makes the goats stiffen when they get excited or hear loud noises.

#AMOPharma Completes Meeting with U.S. FDA and Outlines Plans to Advance Clinical Development of AMO-02 ( #tideglusib ) in Treatment of #MyotonicDystrophy and will conduct a Phase 3 clinical trial in adults living with DM1. Read their full press release at prn.to/3WkVmk0

ICYMI:

PGN-EDODM1 is currently being evaluated in the phase 1 FREEDOM-DM1 trial, a randomized, placebo-controlled, single-ascending dose study.

PepGen #MyotonicDystrophy #AntisenseOligonucleotide

Read more:

cgtlive.com/view/aso-fast-…

Email Your Senators to Sign Senator Amy Klobuchar $10 Million #MyotonicDystrophy Research Funding Letter by May 6th! Your voice is critically important & contacting your Senators takes less than 2 min! You can help change the future of DM research forever: votervoice.net/Myotonic/Campa…

PGN-EDODM1 is currently being evaluated in the phase 1 FREEDOM-DM1 trial, a randomized, placebo-controlled, single-ascending dose study.

PepGen #MyotonicDystrophy #AntisenseOligonucleotide

Read more:

cgtlive.com/view/aso-fast-…

One thing that maybe getting lost here is the terrible pass from RSL too. It was so bad, Thommy couldn’t believe his eyes and the sheer excitement of it all led to him turning into a Myotonic Goat right in front of our eyes.

Figen These are Myotonic or 'Fainting Goats'. They have a disorder that cause their muscles to stiffen when they hear loud noises or get excited.

The 2024 MDF Regional Conference in Houston, TX Starts Off Strong! Over 130 attendees are joining us to unite our local #myotonicDystrophy local communities. Learn more about this 2024 MDF Regional Conference at: myotonic.org/mdf-regional-c…

Ovid Therapeutics keeps an internal list of upcoming readouts, which currently includes uniQure’s phase 1/2 results for its Huntington’s candidate AMT-130, and a data set from Harmony Biosciences mid-stage study of pitolisant in myotonic dystrophy type 1
Meagan Parrish
pharmavoice.com/news/neuroscie…

French guidelines: Expert opinion on mexiletine treatment in adult patients with myotonic dystrophy pubmed.ncbi.nlm.nih.gov/38677940/