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 »
miRecule, Inc., is developing a novel treatment for FSHD
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 »
The Fierce Urgency of Now
“We are now faced with the fact that tomorrow is today. We are confronted with the fierce urgency of now.” Thus Dr. Martin Luther King Jr. exhorted the nation at the March on Washington on that historic summer day in 1963. While King spoke of long-deferred dreams of equality, today in the FSHD community we… Read More »
HAT Trick to Block the DUX4 Gene
Researchers Identify a New Compound that Inhibits the Activity of DUX4 By Alec DeSimone, PhD University of Massachusetts Medical School Led by a team at the University of Minnesota, Minneapolis, researchers have identified a new compound that inhibits the activity of DUX4, implicated as the root cause of facioscapulohumeral muscular dystrophy (FSHD). The compound provides… Read More »
Patient-Focused Drug Development Meeting for FSHD
The FSHD Society announced today that the U.S. Food and Drug Administration (FDA) has approved its application to hold an externally led Patient-Focused Drug Development (EL-PFDD) meeting on facioscapulohumeral muscular dystrophy. This “voice of the patient” meeting, a key component of the Society’s Therapeutic Accelerator initiative, has been scheduled for April 21, 2020, in College… Read More »