by Jennifer Egert, PhD, New York City After being involved with the FSHD Society for more than 10 years, participating in fundraisers, social media, and events, I finally made it to the FSHD Connect meeting in 2018 in Las Vegas. It’s sort of ridiculous that I hadn’t gone until then, especially when the Society is… Read More »
FSHD Connect is a game changer
Which symptoms have the biggest impact on your life?
On April 21, the FSHD Society is convening its landmark Voice of the Patient Forum, our community’s opportunity to testify to the FDA on the impact FSHD. An important question to answer is, which symptoms have the biggest impact on your life? Your testimony will result in a Voice of the Patient Report that will… Read More »
Losmapimod receives orphan drug designation
Fulcrum Therapeutics has announced that losmapimod has received orphan drug designation from the U.S. Food and Drug Administration. Losmapimod is currently in a Phase 2 clinical trial for facioscapulohumeral muscular dystrophy (FSHD). The drug qualified for this status because FSHD, with an estimated 20-40,000 affected individuals in the U.S., is considered a rare disease. (The… Read More »
Why Your Voice Matters
This April 21, the FSHD Society is holding its landmark Voice of the Patient Forum on drug development for FSH muscular dystrophy. You will be hearing a lot about it over the coming months. This event is our community’s opportunity to testify to the FDA on the impact FSHD has on your life and the… Read More »
Voice of the Patient Forum [VIDEO]
Recorded on January 20, this informational video discusses the upcoming Voice of the Patient Forum (or patient-focused drug development meeting) for facioscapulohumeral muscular dystrophy (FSHD) that is being organized by the FSHD Society for April 21. James Valentine explains the role of the patient’s voice in the FDA‘s consideration of drug approval applications. June Kinoshita… Read More »