Clinical trials

UK FSHD patient registry publishes overview of ongoing clinical trials
Ben Porter, curator of the UK FSHD Patient Registry, has published a visual overview of ongoing clinical trials for FSHD (type 1 and 2) worldwide. In addition to the already known Fulcrum Therapeutics trial, which is in clinical phase 2 and whose first results are expected in the middle of the year, you will also find a number of promising gene and other therapies from various companies (e.g. Dyne Therapeutics, miRecule Inc., Myocea Inc., Facio Therapies) and research institutes (e.g. Nationwide Children’s Hospital, King’s College London). If you want to know more about a specific study, you can click on the links provided in the last column.

Facio Therapies announces the completion of real world data study
https://www.facio-therapies.com/news/facio-announces-completion-of-real-world-data-study-in-fshd/

The general overview of the current clinical trials for FSHD https://www.clinicaltrials.gov/ct2/results?cond=FSHD&term=&cntry=&state=&city=&dist=

Fulcrum initiated a Phase 2b clinical trial of losmapimod in August 2019 (https://www.fulcrumtx.com/therapeutic-focus/fshd/)

Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve) (https://bmcneurol.biomedcentral.com/articles/10.1186/s12883-019-1452-x)

ABCs of Clinical Trials with Rabi Tawil MD
Watch the movie on Youtube