FSHD European Trial Network – Virtual Meeting
FSHD European Trial Network: organized by FSHD Europe, with Nicol Voermans as chair, George Padberg as advisor and a core group of FSHD experts.
FSHD is on the doorstep of clinical therapeutic trials, there is interest in FSHD from several pharmaceutical industries and a few trials are currently performed. The guidelines for clinical trials, pharma regulation and participation, and health care provisions in European countries differ in various subtle ways and would benefit from an overall strategy specifically catered to the European situation.
In order to offer the FSHD patient community in Europe the best position in discussions with clinical and basic science researchers and with pharma, FSHD Europe has initiated the FSHD European Trial Network. The network organizes two virtual meetings in Spring 2021 with the following aims:
· Establish the foundation of European FSHD Trial Network
· Increase the commitment of clinicians and researchers in clinical research in Europe
· Harmonize criteria for clinical and genetic diagnosis, for registries and outcome measures
· Exchange of clinical experience and genetic reference material
· Bring Europe on a par with the USA on trial-readiness in FSHD organized
· Engage Pharma and EMA for a Europe wide collaboration
· Harmonize treatment and care for all European FSHD patients
This will be done in collaboration with the Clinical Research Trial Network, TreatNMD and the European Reference Networks for Rare Diseases.