$133,254 awarded to FSHD Clinical Trials Research Network

The FSH Society has awarded a grant for a second year of support for the FSHD Clinical Trials Research Network (CTRN), through the coordinating center based at the University of Kansas Medical Center, Fairway, Kansas. In awarding the grant, the Society notes the value of having a standing, research and trial-ready infrastructure for FSH muscular … Read more of “$133,254 awarded to FSHD Clinical Trials Research Network” »

Job opening: Postdoctoral researcher for Sverdrup lab

Fran Sverdrup, PhD, at the Saint Louis University School of Medicine is recruiting a qualified scientist for the position of POSTDOCTORAL FELLOW in Muscular Dystrophy Drug Discovery. OBJECTIVES: The Postdoctoral Fellow will be part of a team focused on target identification and validation in facioscapulohumeral muscular dystrophy (FSHD). He/she will apply expertise in cell and molecular biology to … Read more of “Job opening: Postdoctoral researcher for Sverdrup lab” »

2018 FSHD Connect Conference registration is now open!

Our community’s premier networking event for immersive learning and community building Our biennial FSHD Connect Conference is an amazing opportunity to meet hundreds of patients and families, as our leading thinkers share the latest in medical management of FSHD and exciting advances toward treatments. Breakout sessions will cover how to cope with practical and emotional … Read more of “2018 FSHD Connect Conference registration is now open!” »

Facio selects first series of potential drug development candidates

We are sharing this news release from Facio, the Netherlands-based biotech founded by FSHD advocates and business leaders with the sole mission of developing treatments for FSHD. In this story, Facio announces that it has identified drug-like molecules that show promise in repressing DUX4, the gene believed to be a key driver of the disease. … Read more of “Facio selects first series of potential drug development candidates” »

Acceleron Announces Preliminary Results from Part 1 of the ACE-083 Phase 2 Trial in Patients with Facioscapulohumeral Dystrophy

We are sharing the following press release from Acceleron Pharma, Inc., regarding the progress of their Phase 2 clinical trial of ACE-083. The FSH Society collaborates with Acceleron to help the company understand patients’ symptoms, motivations, desired outcomes, etc.  – Mean total muscle volume increases of over 12% in the tibialis anterior and biceps brachii … Read more of “Acceleron Announces Preliminary Results from Part 1 of the ACE-083 Phase 2 Trial in Patients with Facioscapulohumeral Dystrophy” »

Video: Molecular therapy for FSH muscular dytrophy

In this FSH Society webinar, Scott Q. Harper, PhD, of Nationwide Children’s Research Institute describes FSHD genetics and explains how gene and molecular therapy could be used to block the genetic mechanism that is thought to cause muscle degeneration in facioscapulohumeral muscular dystrophy. The talk is very understandable to non-scientists and conveys the real sense … Read more of “Video: Molecular therapy for FSH muscular dytrophy” »

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” »