aTyr Pharma releases results of early-onset FSHD trial

SAN DIEGO, April 24, 2017 (GLOBE NEWSWIRE) — aTyr Pharma, Inc. (Nasdaq:LIFE), a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address severe, rare diseases, today announced promising clinical results from its Phase 1b/2 003 trial assessing the safety and potential activity of Resolaris™ in patients with early onset facioscapulohumeral muscular … Read more of “aTyr Pharma releases results of early-onset FSHD trial” »

Acceleron’s ACE-083 clinical trial is now open

Cambridge, Massachusetts-based Acceleron Pharma, Inc., is now recruiting adults with FSH muscular dystrophy for a Phase 2 clinical trial of ACE-083. The initial trial site is located in St. Louis, Missouri. Additional sites across the U.S. are expected to begin recruiting soon. ACE‐083 is an investigational drug that inhibits selected proteins in the transforming growth … Read more of “Acceleron’s ACE-083 clinical trial is now open” »

aTyr Pharma Receives FDA Fast Track Designation for Resolaris™

— First Reported Fast Track Designation for a FSHD Treatment — SAN DIEGO, Oct. 24, 2016 /PRNewswire/ — aTyr Pharma, Inc. (Nasdaq: LIFE), a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address severe, rare diseases, today announced that its product candidate Resolaris™ was granted Fast Track designation by the US Food … Read more of “aTyr Pharma Receives FDA Fast Track Designation for Resolaris™” »

FSH Society Awards Grant to Establish Clinical Trial Research Network for FSHD

(PRWEB) JUNE 14, 2016 The FSH Society, a nonprofit, patient-driven organization that supports research and education for individuals with facioscapulohumeral muscular dystrophy (FSHD) and their families, today announced that it has awarded a $121,000 grant to co-principal investigators Jeffrey Statland, MD of the University of Kansas Medical Center, and Rabi Tawil, MD of the University … Read more of “FSH Society Awards Grant to Establish Clinical Trial Research Network for FSHD” »

Celebrate World FSHD Day on June 20

On June 20, we will celebrate the inaugural World FSHD Day to raise awareness for Facioscapulohumeral muscular dystrophy. Participants from across the globe, including individuals with FSHD, their families and supporters, and advocacy organizations, will come together in various activities to raise awareness for FSHD, one of the most prevalent forms of muscular dystrophy.

FSH Society submits testimony to U.S. Congress

FSH Society President & CEO Daniel Perez has submitted his annual testimony to the U.S. House Appropriations Committee, Subcommittee on Labor, Health and Human Services, Education and Related Agencies (LHHSE). This year, the Society has requested $24 million FY2017 appropriations for NIH research on FSHD.  Identical testimony was submitted to the Senate. The following table from the … Read more of “FSH Society submits testimony to U.S. Congress” »

Publication highlights release of muscular dystrophy action plan

The Muscular Dystrophy Coordinating Committee (MDCC), a congressionally authorized group of representatives from federal agencies and patient advocates, recently released an updated version of its Action Plan for the Muscular Dystrophies. FSH Society played a key role in the formation of the MDCC through its sustained advocacy work with the federal government, and Daniel Perez, … Read more of “Publication highlights release of muscular dystrophy action plan” »