Clinical Trials and Drug Approval

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, 

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.

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

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

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

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.

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.

MDF community members reported on the impact of myotonic dystrophy on the brain from their individual perspectives as people living with DM1, DM2 and as caregivers. Additional insights and comments were provided by members of the conference audience. The session input will be published and used to help drug developers understand the impact of DM on the brain from the patient perspective, and begin to identify clinical trial endpoints. From the 2017 MDF Annual Conference.