Category Archives: Press releases

FSH Society Awards $541,133 in Funding for FSH Muscular Dystrophy Research

Boston-based Non-profit Awards New Grants to Facilitate Search for a Cure

BOSTON – April 5, 2017 – Today the FSH Society, a world leader in combating facioscapulohumeral muscular dystrophy (FSHD), announced it has committed $541,133 in funding to five research projects that aim to break new ground in the search for a treatment and cure for FSHD. These grants follow the Society’s record breaking $1.36 million awarded in total research funding in 2016.

“These grants are a testament to the dedication of researchers within the FSHD community committed to understanding and solving how FSHD works through high quality peer-reviewed research” said Daniel Perez, President and CEO of the FSH Society. “With these grants we look to build upon our record-breaking success in 2016, which would not have been possible without the generosity and sustained support of donors, Society management and staff, our Board members and volunteers.”

The following proposals submitted in August 2016 were approved: Continue reading

FSH Society honors leaders in science and advocacy

Dr. Silvere van der Maarel accepts 2017 FSH Society Pioneer as Michael Altherr looks on.

Dr. Silvere van der Maarel accepts 2017 FSH Society Pioneer as Michael Altherr looks on.

On November 11, the FSH Society honored four distinguished researchers and community members at their inaugural CureFSHD National Gala in Boston, Mass. The event, hosted by WBUR’s Morning Edition Host Bob Oakes, celebrated 25 years of progress towards finding a cure for Facioscapulohumeral Muscular Dystrophy (FSHD). The gala, which attracted more than 250 researchers, scientists, FSHD patients and supporters, was considered a great success by attendees and organizers alike. Continue reading

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 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.”

Continue reading

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

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

American Ninja Warrior features our Ambassador

Tune in on Wednesday, June 8, for American Ninja Warrior on NBCThe episode airing at 8:00pm ET / 7:00pm CT focuses on the popular show’s Atlanta trials and features Spencer Wyckoff, brother of FSH Society Ambassador Carden Wyckoff. He’s competing in the grueling competition for his sister, to raise awareness of FSH muscular dystrophy. Spencer says his sister’s experience with FSHD has made her who she is; organized, motivated, a force for change and a huge advocate in the wheelchair community. He credits Carden with strengthening his family’s bond when she was diagnosed with FSHD at the age of 9 years old. We are so proud of them both!

Please pay tribute to Spencer and Carden’s heroic efforts by making a gift in their honor here.  

Tune in to NBC on Wednesday, June 8 at 8:00pm EST and check out our Facebook and Twitter. Post and tweet about Spencer’s involvement and effort to raise awareness of FSHD using the hashtags #FSHDninja, #ShowYourStrength and #CureFSHD. Please retweet, share and encourage friends to tune in!

Read our press release here.

Dogs destined to develop muscular dystrophy evade their genetic fate

SAB-LouKunkelGolden retrievers with the Duchenne mutation show that disease-modifying genes may provide routes to treat a genetic disorder; possible parallels to facioscapulohumeral muscular dystrophy.

Research led by Louis M. Kunkel, PhD, a member of the FSH Society’s board of directors and scientific advisory board, demonstrates that a disease might be treated by finding a gene that modifies the devastating impact of the mutant gene causing the disease. Continue reading

Gene Interference Technology Used Against FSH Muscular Dystrophy Genetic Defect

dCas9 figProof-of-principle study is the first to use CRISPR technology on the “repeat genome,” as well as its first successful use in primary human muscle cells

Gene interference technology used in FSH muscular dystrophy

Proof-of-principle study is the first to use CRISPR technology on the “repeat genome,” as well as its first successful use in primary human muscle cells

BOSTON – (November 3, 2015) – The FSH Society, the award-winning non-profit and global leader in the quest to cure Facioscapulohumeral Muscular Dystrophy (FSHD), announced that an FSH Society-funded research team led by Peter Jones, PhD, at the University of Massachusetts Medical School (UMMS) has successfully used a derivation of the CRISPR-based gene-editing method known as dCas9 to target and silence the DNA sequence implicated in FSHD. This genetic condition, which affects an estimated 1 in 8,000 people, is among the most common forms of muscular dystrophy. Continue reading

FSH Society Beneficiary of Variety of Fall Fundraising Events

logoPRWebFrom PRWeb

The FSH Society, the Massachusetts-based charity that has transformed the science of the little-known and incurable disease, facioscapulohumeral muscular dystrophy (FSHD), will be the beneficiary of a collection of fundraising events this fall. From Boston to Los Angeles, each event has the same mission – to raise awareness of the FSH Society and further FSHD research. Continue reading

First-Ever FSHD Evidence-based Care Guideline Published by the American Academy of Neurology

From PRWeb

Contact Information:
Rosie Hale
617-779-1893
FSHSociety@shiftcomm.com

Guideline is a landmark step forward for the diagnosis; comes as a result of FSH Society’s advocacy efforts over the past 20 years

BOSTON, MA (July 27, 2015) – The FSH Society, the Massachusetts-based charity that has transformed the science of the little-known and incurable disease, facioscapulohumeral muscular dystrophy (FSHD), today announced the introduction of the first-ever care guideline for FSHD by the American Academy of Neurology (AAN). The FSHD care guideline is published today. Continue reading