FSHD Patient Advocacy
Patient advocacy is an important part of helping others find a treatment and perhaps ultimately a cure.
20/11/2023
Join us tonight.
Young Adults - Clinical trial updates | FSHD Society 8:00 pm ET | 7:00 pm CT | 6:00 pm MT | 5:00 pm PT Join us as June Kinosh*ta, Senior Director of Research & Education, answers your questions about... Read More »
18/09/2023
Dr. Rabi Tawil FSHD Research & Retirement Interview 09/07/2023 Dr. Rabi Tawil is one of three driving forces for creating the circumstances of Facioscapulohumeral dystrophy (FSHD) research for understanding of the diseas...
16/01/2023
New therapeutic in the pipeline.
05/07/2022
Fulcrum enrolled the first of 230 in REACH, its phase 3 trial of losmapimod.
I am continuing my participation in Fulcrum’s OLE of its phase 2 study as stage 3 formally begins.
Fulcrum Therapeutics Enrolls First Patient in Pivotal Global Phase 3 Clinical Trial of Losmapimod for Facioscapulohumeral Muscular Dystrophy (FSHD) – REACH is the first Phase 3 trial for this rare, progressive, and debilitating muscular disease –– Losmapimod is an oral small molecule that has the...
15/04/2022
Not letting FSHD stop me from achieving a life long ambition, diving the Great Barrier Reef.
If you have desires to achieve life goals, go for it! It may take some planning and some work to overcome your mobility limitations, but it’s worth it.
Don’t left FSHD define your life!
Live it to the fullest possible and enjoy it! 👍
08/03/2022
FSHD research is about to get a huge boost of support with resources that could really make a difference.
Chip Wilson commits $100 million to fight the disease FSHD The Lululemon founder has what's known as facioscapulohumeral muscular dystrophy (FSHD) and foresees one day needing to use a wheelchair.
04/03/2022
Good news on the FSHD front!
Fulcrum is going for phase 3 of losmapimod.
Fulcrum plans phase 3 trial of losmapimod to treat rare muscle disorder Fulcrum Therapeutics (FULC) plans to begin a phase 3 trial, called REACH, of losmapimod in people with facioscapulohumeral muscular dystrophy ((FSHD)) in Q2 2022.FSHD is a rare...
The trial did not meet its primary endpoint.
However, this was a theoretical target and DUX4 is notoriously difficult to measure.
On the important clinical measurements there was a noted reduction of muscle fat infiltration and some improvements in muscle function or at least no loss relative to placebo in any of the group of the genuine drug.
Placebo group tended to lose muscle function but none apparently on losmapimod which were secondary endpoints.
The theoretical primary endpoint is going by the wayside. The actual clinical outcomes such as strength maintaining or improvement coupled with improvements in functional reachable workspace outcomes may then become its revised primary endpoints (ie; the important functional ones).
Now that results have been made public I can share my experience as having been in the study.
My experience has been either no progression or slight improvement.
Given the variability of progression of the disease my results appear consistent with the conclusion by Fulcrum. No deterioration or a little improvement.
I am expecting they are going to move the study for confirmatory trial under a fast-track FDA approval process.
It’s going to be up to the FDA if phase III will be green lighted. Not clear if the OLE of the phase iib will be continued at this point. I haven’t been notified to this as of yet but should soon.
I have the distinct privilege of sitting down with Dr. Rabi Tawil to discuss topics related to FSHD. Dr. Tawil is a pioneer in the research of the underlying mechanisms of FSHD and is also regarded as one of the very best clinicians worldwide in the field of FSHD and other neuromuscular diseases.
In 2010, Dr. Tawil in collaboration with Drs. Silvere van der Maarel and Stephen Tapscott published their Unifying Theory of FSHD that identified the underlying mechanisms of the disease, its type 2 variant, and finally gave Pharma the therapeutic target (DUX4) and disproved the idea that the FRG1 gene was responsible as had been suggested from previous research.
This collaboration reinvigorated research that had otherwise been on the decline because of the obscurity of the disease. No work has been more influential in moving the field towards a treatment or cure.
Dr. Tawil will answer questions from participants, so we hope that you will join us for this special event.