Clinical Trials and Drug Approval

Dyne Therapeutics - Meet the DM Drug Developers

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.

AMO Pharma - Meet the DM Drug Developers

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.

Industry Updates and Q&A - 2019 Myotonic Annual Conference

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.

Ionis Pharmaceuticals - Industry Updates On Drug Development (2018 MDF Annual Conference)

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

Living with DM: Patients Report on Changes Over Time (2018 MDF Annual Conference)

This interactive panel session was presented in a similar format to the Patient-Focused Drug Development meeting presented by MDF as a part of the 2016 MDF Annual Conference, and the CNS Endpoint Development discussion presented at the 2017 MDF Annual Conference.

AMO Pharma Ltd. - Industry Updates On Drug Development (2018 MDF Annual Conference)

Joseph P. Horrigan, MD, from AMO Pharma Ltd. provides an update on their drug development efforts in the DM field. 

Expansion Therapeutics - Industry Updates On Drug Development (2018 MDF Annual Conference)

Representatives from biotech and pharma companies will provide updates on their drug development efforts in the DM field. Matthew Disney, PhD, Expansion Therapeutics

Locana - Industry Updates On Drug Development (2018 MDF Annual Conference)

Ranjan Batra, PhD, Locana, provides an update on their drug development efforts in the DM field. 

Accelerating The Search For Therapies: What's Happening, What's Next (2018 MDF Annual Conference)

Dr. Elizabeth Ackermann reports on the results of the 3-year MDF and Wyck Foundation-funded drug development acceleration effort, MDF 3.0, and introduces MDF’s new initiative, MDF 4.0, including the $1M gene editing project, the Myotonic Dystrophy Clinical Research Network study and other aggressive therapy development programs.

Bringing the Patient Voice to CNS Targeting Drug Development 2: Industry Observations and Feedback

Video #2 from the session: Bringing the Patient Voice to CNS Targeting Drug Development in DM. Dr. Gersham Dent, a neuroscientist working on the DM1 drug development program at Biogen, provides observations on panelists' CNS symptom experiences with regard to possible endpoint identification for future clinical trials of CNS-targeting therapies. From the 2017 MDF Annual Conference.

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