At the end of the day, if there were a drug that stopped FSHD in its tracks, would you want it, even if it did not restore what you have already lost? What if a drug made you stronger temporarily, even if it did not stop the disease? Only those who are directly affected by… Read More »
The Future is Now for the Voice of the Patient
#GivingTuesday Telethon
#GivingTuesday is a global day of giving fueled by the power of social media and collaboration. This year, it falls on December 3, and we are marking the day by holding our first GivingTuesday Telethon. Streamed over Facebook Live, our Telethon host will interview leaders in our community—activists, celebrities, scientists, doctors, people doing awesome things—to… Read More »
The Future is Now for Natural History Studies
Here’s what keeps us up at night: researchers will find a drug that actually works, and yet without understanding the natural history of the disease, they can’t prove the drug is what made the difference. Therefore, the drug efficacy is difficult to prove to the FDA. The very first question a drug company asks when… Read More »
$447,620 in funding for four new projects
The FSHD Society board of directors approved $447,620 in funding for four projects submitted in the February 2019 cycle. These grants will move forward four distinct strategies that show promise. Identification and testing of DUX4 inhibitory compounds. Angela Lek, PhD, Yale University, Hartford, Connecticut, USA, and Louis Kunkel, PhD, Boston Children’s Hospital, Massachusetts, USA. $110,000… Read More »
Finding strength in our pain
The suffering that FSHD has caused has also made us stronger people By Meredith Huml Angier, North Carolina The words “no cure” and “no treatment” were dropped casually into my lap after much prodding and poking and examination on the day I was diagnosed with a disease I’d never heard of. I was 12… Read More »