Expansion Therapeutics

    Drug #1: ERX-963

    Approach: Unknown

    Status: In June 2019 Expansion commenced a Phase 1 trial in 12 adult-onset myotonic dystrophy patients.  Trial failed to show efficacy.  See AAN 2021 presentation.

    

     Drug #2: Small molecule RNA inhibitor

     Status: Preclinical

     Company Website with description of approach

     Video from 2018 MDF Conference

     January 3, 2018 Press Release on Expansion's launch 

     Article on preclinical tests of Expansion's DM1 drug

     Publication of preclinical tests

     September 14, 2019 Presentation at Myotonic Conferenct (1:00)

     Patent Application:  MODULARLY ASSEMBLED SMALL MOLECULES FOR THE TREATMENT OF MYOTONIC DYSTROPHY TYPE 1

     April 2020 presentation by Dr. Disney: Advancing Precision Medicines. Discussion of myotonic dystrophy at 41:50 and 1:05:00

     April 2021 presentation for Myotonic Dystrophy Foundation