An undeniable sense of optimism

In this interview, Dr. Jeffery Statland of the University of Kansas Medical Center explains how FSH Society funding seeded the creation of the seven-site Clinical Trial Research Network (CTRN), which in turn led to the largest clinical trial preparedness study that has ever been run in the United States. We have entered a convergence zone―a … Read more of “An undeniable sense of optimism” »

Intriguing research on tyrosine kinase inhibition as a potential therapy for FSHD: Sunitinib rescues muscle cells’ ability to develop

Written by Jim Albert Eldersburg, Maryland   A cancer drug has been shown to potentially rescue some of the damaging effects of DUX4, the gene implicated in FSH muscular dystrophy. The laboratory of Peter Zammit, PhD, Randall Division of Cell and Molecular Biophysics, King’s College London, United Kingdom, in collaboration with Robert Knight, PhD, of … Read more of “Intriguing research on tyrosine kinase inhibition as a potential therapy for FSHD: Sunitinib rescues muscle cells’ ability to develop” »

Q &A on potential use of BET inhibitors for FSHD

On February 13, Canadian biotech, Reserverlogix announced that facioscapulohumeral muscular dystrophy (FSHD) is one of two new indications it is pursuing involving its lead drug, apabetalone (RVX-208) which inhibits bromodomain and extra-terminal (BET) epigenetic readers.  It mentioned research conducted at Saint Louis University demonstrating apabetalone mediated modulation of important targets in FSHD.  The FSH Society … Read more of “Q &A on potential use of BET inhibitors for FSHD” »

Johns Hopkins Studies

Johns Hopkins and the Kennedy Krieger Institute are currently recruiting for two studies!  Volunteering for studies like this helps provide researchers with the information they need to provide better treatments, understand the mechanisms of the disease, and search for a cure. For Family Members (no travel necessary!): The Johns Hopkins Hospital and the Kennedy Krieger … Read more of “Johns Hopkins Studies” »