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 factor‐beta (TGF‐β) superfamily involved in the regulation of muscle size and strength. ACE‐083 has been designed to increase muscle size and strength specifically in the muscles into which the drug is administered. Acceleron is developing ACE‐083 for diseases in which improved muscle strength in a specific set of muscles may provide a clinical benefit to patients, such as FSHD. As an “investigational” agent, ACE‐083 is not approved by any regulatory agency for use in any country.
For full details of the clinical trial, including updates on new clinical trial locations, please visit ClinicalTrials.gov: Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD).
Additional details can be downloaded here:
Produced for the FSH Society’s 25th anniversary, our new documentary tells the story of how the Society’s work has led to the discovery of the genetic cause of facioscapulohumeral muscular dystrophy and brought patients out of the darkness into the light. We premiered the film at our CureFSHD gala on November 11.
To all the patients, families and friends who have supported the Society through the years, this is your story. Please share this with others to help them understand what FSHD is, the great progress that has been made, and how their support can lead to a brighter future for tens of thousands of patients.
— 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 and Drug Administration (FDA) for the treatment of facioscapulohumeral muscular dystrophy (FSHD), making it the first known therapeutic candidate for the treatment of FSHD to receive the designation. Resolaris, a designated Orphan Drug in FSHD, is currently being studied in a Phase 1b/2 clinical program.
“This Fast Track designation, which is granted to drug candidates addressing serious conditions and that demonstrate the potential to address unmet medical needs, represents another step forward for our rare muscle disease franchise,” said John Mendlein, PhD, CEO of aTyr Pharma. “This designation highlights the significant need to develop and ultimately approve meaningful new therapeutics to treat patients with rare myopathies, such as 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 of Rochester Medical Center, to expedite the development of new therapies for FSHD by developing a core FSHD Clinical Trial Research Network (CTRN). The FSH Society is funding four institutions that are piloting the CTRN: the University of Rochester, Kennedy Krieger Institute, Ohio State University, and the University of Kansas, all established centers with expertise in FSHD and neuromuscular clinical trials. Continue reading
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. Continue reading
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 FSH Society’s testimony shows the progress being made in NIH funding for FSHD projects.
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, President and CEO of the Society, serves on the MDCC as a voice for the FSHD community.
Aerial view of NIH campus.
We have some good news to share on National Institutes of Health funding for FSHD. As part of the FSH Society’s committee work in Washington, DC, we recently received FY2015 data release on NIH support for the muscular dystrophies and were very pleased to see actual 2015 fiscal year NIH funding support for FSHD research at $8,397,995. We thought you would be too. Continue reading
Fran Sverdrup with his daughter Naia
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Saint Louis University’s (SLU) Center for World Health and Medicine, announced that they have entered into three-year agreement to collaborate on the development of small molecule therapeutics for the potential treatment of Facioscapulohumeral Muscular Dystrophy (FSHD). Continue reading
Did you hear? The FSH Society has been awarded a grant by Staples Foundation. We plan to use the donation to support public awareness and youth education about FSHD, such as a social media awareness raising scholarship competition administered by the Jain Foundation.
Eligible students can compete this spring for the scholarship by posting a fact and image about FSHD. If you know a student who would be interested in this competition, please spread the word about this opportunity. For more information on how to submit, visit: http://www.jain-foundation.org/scholarship/social-media. Continue reading