Category Archives: FSHD Research

FSHD Canada awards $20K for FSH Society research grant

Yi-Wen Chen PhD

The FSH Society is pleased to announce that it has received a grant of $20,000 from the FSHD Canada Foundation in support of an exciting, treatment-focused project, “Developing LNA-based therapy for facioscapulohumeral muscular dystrophy.”

 “We are delighted to help Neil Camarta and his colleagues at FSHD Canada help make a huge impact on FSHD by helping to fund high-quality research, both critical and necessary—in Canada and around the globe,” said Daniel Perez, President, CEO and CSO of the FSH Society. “Projects as this one are ideal for achieving the purposes of both our organizations and helping to provide insights and hope for the constituents we serve.”   Continue reading

Clinical Trial Research Network receives major award from NIH

by June Kinoshita 

Dr. Jeffrey Statland

The FSHD Clinical Trial Research Network (CTRN), which was launched last year with a $121,000 grant from the FSH Society, has received a UO1 award from the National Institutes of Health. The project will receive $469,642 for the first year. Additional funds, up to a total of nearly $2.7 million, will be released upon reaching annual milestones. The principal investigators are Rabi Tawil, MD, of the University of Rochester, and Jeffrey Statland, MD, of the University of Kansas.

“The support of the FSH Society and a private donor helped us establish the infrastructure necessary to propose such a study, and the continuing support will be essential not just for this study, but for future efforts toward creating new outcome measures, developing new therapies, and training the next generation of FSHD researchers,” said Statland. Continue reading

FSH Society awards over a half million dollars for new research

The FSH Society, a world leader in combating facioscapulohumeral muscular dystrophy (FSHD), announced this spring that it has committed $550,792 in funding to six 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 for 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, CEO & CSO of the FSH Society. “With these grants we look to further increase our understanding of the inner workings of FSHD and build upon our 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.” Continue reading

FSH Society Talk Radio with Dr. Charis Himeda

Charis Himeda, PhD.

UPDATE: Our show has been rescheduled for Wednesday, August 30, 2017, 9:00 pm EST / 8:00 CST. Visit us here for updates. Listen live or via podcast HERE!

Our guest is Charis L. Himeda, PhD, Research Assistant Professor at the Center for Molecular Medicine at the University of Nevada, Reno School of Medicine. She has worked in the fields of myogenesis and muscle disease for many years, and is currently using cutting-edge technologies to examine mechanisms of epigenetic dysregulation in FSHD muscle cells and explore potential avenues of therapy.  Continue reading

Why I donated tissue

by MAUREEN EYE Centerport, New York

In November of 2016, I underwent scapula fusion surgery in Baltimore, Maryland. During that surgery I had the privilege to donate muscle tissue for FSHD research.

The donation itself was relatively simple. The paperwork prior to the surgery was minimal and mainly taken care of by the doctor’s office. The procedure itself was included as part of my overall surgery, and did not cause any further pain or loss of mobility.

Several months prior to the surgery, June Kinoshita from the FSH Society reached out to me to ask if I would be interested in donating muscle tissue taken from my surgery. It wasn’t even a question in my mind. Without hesitation I committed to donating muscle. Continue reading

From our CEO, a call to FSHD researchers

Dear Research Colleagues,

The late Senator Paul Wellstone of Minnesota

As you know, the FSH Society constantly encourages the National Institutes of Health (NIH) to increase the number of grants and the number of dollars it funds on one of the most common forms of muscular dystrophy–facioscapulohumeral muscular dystrophy (FSHD)–while urging the FSHD research community to help the NIH to achieve higher levels of spending on FSHD by submitting grant applications. In fiscal year 2017, the NIH estimates funding $80 million on all forms of muscular dystrophy (source: NIH online Research, Condition, and Disease Categorization (RCDC) system). Continue reading

FSH Society grant award for myostatin study

Belgian Blue cattle are ripped, thanks to a mutation in their myostatin gene. Photo credit: https://commons.wikimedia.org/w/index.php?curid=1702186

In April, the FSH Society awarded Julie Dumonceaux PhD, or University College London, Institute of Child Health. a grant of $9,659.43 for one year for a project aimed at better understanding patients’ response to a class of drugs called myostatin inhibitors.

These drugs, such as Acceleron’s ACE-083 which is currently in a clinical trial in FSH muscular dystrophy patients, target myostatin, a molecule that the body produces naturally to inhibit muscle growth. The rationale for blocking myostatin is to enable muscle to achieve greater mass than it otherwise would. In conditions such as FSHD, where muscle mass is lost, a myostatin inhibitor would in theory enable muscle to re-grow without having to fight an uphill battle against the growth-blocking effects of myostatin. Continue reading

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 dystrophy (FSHD).

Read the full press release here.

Intriguing research on tyrosine kinase inhibition as a potential therapy for FSHD: Sunitinib rescues muscle cells’ ability to develop

Written by Jim Albert
Eldersburg, Maryland

 

A cancer drug has been shown to potentially rescue some of the damaging effects of DUX4, the gene implicated in FSH muscular dystrophy. The laboratory of Peter Zammit, PhD, Randall Division of Cell and Molecular Biophysics, King’s College London, United Kingdom, in collaboration with Robert Knight, PhD, of the Department of Craniofacial Development and Stem Cell Biology at King’s, has published the results of its research on the activity of an FDA-approved drug, sunitinib, as having potential therapeutic activity for FSH muscular dystrophy (FSHD). Continue reading

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