friends of FSH Research


Over 10 Years of Creating Hope

The mission of Friends of FSH Research is to accelerate the discoveries that will lead to treatments or cures for FSHD. To achieve our goal, we actively manage a portfolio of research that supports a pipeline for drug discovery. Our board, staff, volunteers, and world-class scientists are fully committed to transparent, reproducible, and transformative science that will inspire hope for people with FSHD. Learn more >>

facebook twitter pinterest instagram

Over $3.85 million

funded to date


Attracted to research

Check Out Your Impact >>

"Hopefully one day FSHD will be stopped in its tracks. I know that there is still a long road ahead, with many milestones to pass but the journey isn’t quite so scary knowing that I am not alone and that there are those willing to keep pushing ahead, guiding the way until we find the key to unlock that final door."

- T. Pilato

Our vision: One day the diagnosis of FSH Muscular Dystrophy will be followed by "...there is a treatment."

Latest News

From Passive to Active

— March 19, 2018

ENMC 25th anniversary summit - The Patient Role in Shared Decision Making.

Spring RFP

— February 7, 2018

Spring 2018 Request for Research Proposals.

Research Presentation Slides

— February 1, 2018

Slide decks for Dr. Tapscott & Dr. Wang from the 27-Jan presentations.

Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD

— January 17, 2018

A publication from Scott Harper's lab reporting on the use of an AAV vector to test the safety of two miRNAs in…

Progress Report: 3rd Gen Oligonucleotide Targeting DUX4 in Mouse Model

— January 1, 2018

Yi-Wen Chen DMV PhD provides an update on investigating 3rd generation oligonucleotides.


Ask the Expert

Therapy Q&A

by Amanda Rickard

Interview of Dr. Joel Chamberlain regarding her lab's work on therapy development.
to