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 »
$447,620 in funding for four new projects
The Future is Now for Clinical Trials
Our community has been promised for decades that “one day in the future” there will be treatments for FSHD, and so it’s amazing to realize that day is almost upon us. The future is NOW for clinical trials. In 2019, we saw two Phase 2 trials—for Acceleron’s ACE-083 and Fulcrum’s ReDUX4 (losmapimod). A dozen biopharma… Read More »
A new, improved CRISPR-Cas9 technology
By Angela Lek, PhD Yale University The discovery of CRISPR-Cas9 gene-editing technology has undoubtedly revolutionized the field of human genetics, enabling for the first time in human history the ability to target disease-causing mutations in our DNA–the root cause of genetic diseases. Now a new, improved version of CRISPR-Cas9 has been developed… Read More »
miRecule, Inc., is developing a novel treatment for FSHD
In their offices in Maryland, just northwest of Washington, DC, miRecule Inc. and its founder Anthony Saleh, PhD, are hard at work developing a novel treatment for FSHD. In the early 2000s, Saleh was a graduate student at Johns Hopkins School of Public Health in Baltimore, Maryland, working on a new class of drugs known… Read More »
Progress Report on Therapeutic Accelerator for FSHD
Last week, Mark Stone, the FSHD Society president and CEO, gave his fourth-quarter progress report on the Therapeutic Accelerator for FSHD treatments. His presentation was given via webinar to leadership donors and key volunteers, including chapter directors and Walk & Roll leaders. The recorded talk and Q&A are now available on the FSHD Society’s YouTube… Read More »