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.
Underscoring the keen interest in creating a network for FSHD clinical research, additional funding obtained by Dr. Tawil from a private foundation will enable the CTRN to be expanded to seven sites. The additional sites include the University of California – Los Angeles, The University of Utah, and the University of Washington.
The CTRN will be composed of academic research centers working closely with the FSH Society to collaboratively develop, test and validate clinical outcome measures and biomarkers. A prospective study of 150 patients followed over 18 months will be needed to validate the outcome measures of the clinical trial research network.
“This Clinical Trial Research Network is a milestone for the FSHD and muscular dystrophy community,” said Daniel Perez, co-founder, president and CEO of the FSH Society. “The network will significantly increase the likelihood that promising therapeutic interventions in FSHD can be translated into clinical trials for patients. We are also thrilled that the CTRN will ensure the patient voice is heard and addressed throughout the drug development process via its Patient Engagement Circles.”
Research conducted by the CTRN includes a longitudinal natural history study, which “will help to establish the variability and rate of progression of FSHD in patients,” said Dr. Jeffrey Statland, Assistant Professor of Neurology at the University of Kansas Medical Center. “This information is crucial in clinical trial design as it allows the estimation of the study sample size and the power to detect a predicted therapeutic effect size.”
The Clinical Trial Research Network members include:
- Rabi Tawil, MD. Professor of Neurology, Pathology and Lab Medicine, and head of the Fields Center for FSHD Research at the University of Rochester Medical Center.
- John Kissel, MD. Professor of Neurology and Chairman of the Department of Neurology at the Ohio State University Medical Center.
- Kathryn Wagner, MD, PhD. Director of the Center for Genetic Muscle Disorders at the Kennedy Krieger Institute and Associate Professor of Neurology and Neuroscience at the Johns Hopkins School of Medicine.
- Jeffrey Statland, MD. Assistant Professor of Neurology at the University of Kansas Medical Center in Kansas City.
- Nicholas Johnson, MD. Assistant professor of neurology, pediatrics, and pathology at the University of Utah
- Perry Shieh, MD, PhD. Associate professor of neurology and director of the neuromuscular fellowship program at the University of California, Los Angeles
- Leo Wang, MD, PhD. Assistant professor in the department of neurology at the University of Washington Medical Center.
FSHD, one of the most prevalent forms of muscular dystrophy, is a degenerative muscle disease that causes progressive weakness, usually starting with the face, shoulder, arms, legs and torso, but may affect almost any skeletal muscle in the body. FSHD affects approximately 870,000 people and there is no treatment or cure, yet. Approximately one-quarter to one-third of patients end up in wheelchairs.
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About the FSH Society: The FSH Society is a world leader in combating muscular dystrophy. The non-profit has provided millions of dollars in seed grants to pioneering research worldwide, creating an international collaborative network of patients and researchers. The FSH Society seeks to serve as a source of information and support for all patients and families with FSHD; act as a driving force in the development of research directed towards treatments and ultimately a cure; and bring support to patients and research for FSHD through effective engagement of governmental and private sector organizations and entities. For eight consecutive years, the Society has received the Charity Navigator’s four-star rating, the highest distinction held by less than two percent of non-profit organizations in the country. The FSH Society offers a community of support, news and information for FSHD patients and families through its website at https://www.fshsociety.org. For more information about FSHD, please contact the Society at 781-301-6649.
June Kinoshita: The FSH Society, 781.301.6649; june.kinoshita(at)fshsociety(dot)org
Theresa Masnik: SHIFT Communications, 617.779.1871; FSHSociety(at)shiftcomm(dot)com