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Last updated on April 24, 2014 at 21:24 EDT

Foundation for Angelman Syndrome Therapeutics (FAST) Funds Human Clinical Trial

February 14, 2012

Potential treatment for Angelman Syndrome to be conducted at the University of South Florida

DOWNERS GROVE, Ill., Feb. 14, 2012 /PRNewswire-USNewswire/ — The Foundation for Angelman Syndrome Therapeutics (FAST) announces it will fund a human clinical trial to assess a potential treatment for the rare genetic disorder, Angelman Syndrome (AS). The Minocycline in the Treatment of Angelman Syndrome study will examine if the off label administration of minocycline will alter the severity of symptoms associated with Angelman Syndrome. Minocycline, an FDA approved antibiotic, is traditionally used to treat bacterial infections in several organ systems.

The single arm open label trial will take place at the University of South Florida. Eligible participants must be between the ages of 4 to 12 years of age and have a molecularly confirmed diagnosis of Angelman Syndrome.

Paula Evans, Founder and Chairperson of FAST, stated, “There are currently no treatments for the cognitive, motor or behavioral deficits associated with Angelman Syndrome. The idea for testing FDA approved drugs in the AS mouse was born in FAST as a way to bring potential therapeutics to use with the greatest speed and efficiency. We could not be more pleased or excited that an FDA approved drug has shown promise in the AS mouse model and may potentially show improvements in children with AS.”

“This study has the potential to play a much-needed role in improving the core symptoms of Angelman Syndrome and helping patients and families achieve an improved quality of life,” said University of South Florida Neuroscientist Edwin Weeber, PhD.

“We are thrilled that we were able to go from testing minocycline in the AS mouse to human clinical trials within one year; a remarkably rapid time frame that speaks to the power of this approach,” said Rebecca Burdine, PhD, Chief Science Officer for FAST. “We remain committed to supporting ongoing research to test additional FDA approved drugs for their potential therapeutic use in treating the symptoms of AS.”

Information about the clinical trial is posted on clinicaltrials.gov and on the FAST website at www.CureAngelman.org. FAST was recently featured on the NBC special, American Giving Awards, when Golden Globe winning actor Colin Farrell received the Leadership Award for his work with the organization. FAST secured the funding for this clinical trial through the Vivint Gives Back project.

About Angelman Syndrome
Angelman Syndrome is a severe neurological disorder characterized by profound developmental delays, epilepsy, and problems with motor coordination (ataxia) and balance. Individuals with AS do not develop functional speech. The seizure disorder in individuals with Angelman Syndrome can be difficult to treat. Feeding disorders in infancy are common, and some persist throughout childhood. Sleeping difficulties are commonly noted in individuals with Angelman Syndrome. AS affects all races and both genders equally and occurs in approximately one in 10,000 to 15,000 births. For more information about Angelman Syndrome, please visit http://www.CureAngelman.org.

About FAST
FAST is a Section 501(c)(3) non-profit research organization narrowly focused on funding research that holds the greatest promise of treating Angelman Syndrome. FAST-Trac Grants and Grants-in-Aid are reviewed on a rolling basis with no application deadline. To learn more about FAST’s mission and funding priorities, or to make a donation towards FAST’s research program visit www.CureAngelman.org.

About USF Health
USF Health’s mission is to envision and implement the future of health. It is the partnership of the USF Health Morsani College of Medicine, the College of Nursing, the College of Public Health, the College of Pharmacy, the School of Biomedical Sciences and the School of Physical Therapy and Rehabilitation Sciences; and the USF Physician’s Group. The University of South Florida is a global research university ranked 34th in federal research expenditures for public universities.

SOURCE Foundation for Angelman Syndrome Therapeutics (FAST)


Source: PR Newswire