Focus on Fulcrum’s FSHD tissue modeling project

Cambridge, Massachusetts, biotech company Fulcrum Therapeutic’s research program on FSH muscular dystrophy was featured in Clinical Leader yesterday. The company “is using tissue donated from patients with facioscapulohumeral (FSH) muscular dystrophy, an incurable form of the disease, to find a treatment,” the article reported. “The tissue is used to create research models that look and respond … Read more of “Focus on Fulcrum’s FSHD tissue modeling project” »

Recent FSHD research publications

Notable findings and advances from the past six months by DANIEL PAUL PEREZ Chief Science Officer, FSH Society Asterisk denotes FSH Society funding acknowledged in paper. “Conservation and innovation in the DUX4-family gene network,” from the laboratory of Stephen J. Tapscott at the Fred Hutchinson Cancer Center in Seattle, Washington (Whiddon et al. Nat Genet. … Read more of “Recent FSHD research publications” »

Placing a BET on blocking DUX4

by FRAN SVERDRUP, PHD St. Louis, Missouri DUX4 is considered to be a key cause of muscle degeneration in FSH muscular dystrophy. This is why we are interested in finding drugs that “turn off” the production of DUX4, which would prevent the many detrimental activities of this toxic protein. Such drugs would potentially provide the … Read more of “Placing a BET on blocking DUX4” »

Nearly $1.2 million committed in 2017 to FSH muscular dystrophy research

The FSH Society has awarded grants totaling $616,467 to seven research projects submitted during the February 2017 grant cycle. This brings the Society’s total new research commitments to $1,167,260 for the year. Reviewed by the Society’s world-class Scientific Advisory Board, these cutting-edge projects will help to accelerate the development of treatments. Michael Kyba’s project will carry … Read more of “Nearly $1.2 million committed in 2017 to FSH muscular dystrophy research” »

A new DUX4 mouse with muscle disease

The FSH muscular dystrophy scientific literature finally has a publication describing a genetic mouse model that develops skeletal muscle disease. This work comes via the laboratory of Michael Kyba, PhD, at the University of Minnesota’s Lillehei Heart Institute. This murine model, called iDUX4 pA, has a number of compelling similarities to the human disease. Ever … Read more of “A new DUX4 mouse with muscle disease” »

Testosterone and human growth hormone clinical trial for FSHD

UPDATED October 30, 2017 Researchers at the University of Rochester in New York are conducting a research study to learn more about a potential symptomatic therapy for FSHD. This study may help determine if a combination of drugs (recombinant human growth hormone [rHGH] and testosterone) can be safely given to patients with FSHD and possibly … Read more of “Testosterone and human growth hormone clinical trial for FSHD” »

An FSHD Antisense Therapy Primer

Q&A With Dr. Yi-Wen Chen by JIM ALBERT, Eldersburg, Maryland Antisense therapy is a form of treatment for genetic disorders. In the past year antisense drugs have been approved by the FDA for the treatment of two types of muscular dystrophies: some forms of Duchenne muscular dystrophy, and spinal muscular atrophy. While antisense therapy for the … Read more of “An FSHD Antisense Therapy Primer” »