Clinical Trials and Drug Approval

Presented on September 10, 2022.

Presented by Jacinda Sampson, MD of Stanford University and Kevin Brennan of Bluebird Strategies

Learn about biobanks, natural history studies, clinical trials, registries, and other types of research, the role that they play in drug development, and how DM1 and DM2 families can participate right now. Hear how MDF has and continues to facilitate research towards a cure, and discover ways that you can help increase federal funding for DM research right now!

Presented on August 5th, 2022. Join Alayna Tress, Dr. Michelle Mellion, Dr. Jaya Goyal, Holly Hand, and Dr. Jane Larkindale from the PepGen team for a presentation on their myotonic dystrophy type 1 program.

Presented on July 22nd, 2022. The Harmony Biosciences team presents on their ongoing clinical trial, "Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1".

Presented on July 1st, 2022. The Avidity Biosciences team presents on their AOC technology, the progress of their AOC 1001 program for DM, and the ongoing MARINA^TM trial.

Presented on June 17th.

Do you have questions for DM experts? Join Sarah Howe, MBA, of the Marigold Foundation for an “Ask-the-Expert” discussion, A Community Portrait through Surveys and Insurance Claims Data! 

Presented on June 3rd, 2022. Dr. Beatriz Llamusi Troisi and Dr. Judy Walker present on Arthex Biotech's lead investigational compound for myotonic dystrophy, ATX-01.

Presented on April 1st, 2022. Dr. Ashish Dugar, SVP and Global Head of Medical Affairs at Dyne Therapeutics, will present an overview of Dyne’s DM1 candidate, DYNE-101, including preclinical data and plans for the upcoming clinical trial. Q&A hosted by Molly White, Vice President, Medical Communications & Advocacy.

In August 2020, AMO Pharma published the results of a Phase 2 study utilizing AMO-02 (Tideglusib) for Childhood Onset DM1 in Pediatric Neurology. AMO Pharma is excited to announce they will be moving forward with the REACH-CDM Clinical Trial for AMO-02 after the US FDA and Health Canada approved their recently revised study protocols.

Representatives from biotech and pharma companies will provide updates on their drug development efforts in the DM field, including companies new to the Myotonic Annual Conference. The industry updates are followed by the Q&A session where questions submitted throughout the conference are answered by a panel of DM Professionals.

Laurence Mignon, PhD, IONIS Pharmaceuticals provides an update on their drug development efforts in the DM field,