The Natural History Study for Rett syndrome is funded for 5 additional years! The University of Alabama at Birmingham received a cooperative agreement U54 award from the National Institute of Child Health and Human Development to conduct this work within the ORDR/NCATS led Rare Diseases Clinical Research Network (RDCRN). Under the leadership of Dr. Alan Percy, the study will involve nine PARTICIPATING sites [UAB – Alan Percy, MD; Baylor College of Medicine - Dan Glaze, MD; Boston Children’s Hospital (Harvard) - Walter Kaufmann, MD; Greenwood Genetics Center – Steve Skinner, MD; Children’s Hospital of Pennsylvania (U. Penn) – Eric Marsh, MD PhD; University of Rochester – Alex Paciorkowski, MD, FACMG; University of Colorado Medical School (Denver) – Tim Benke, MD PhD; University of San Diego – Jeff Neul, MD PhD; Vanderbilt University – Sarika Peters, PhD] and two ENROLLING sites [Rush Medical School – Peter Heydemann, MD; UCSF Benioff/Oakland Children’s Hospital – Mary Jones, MD]. This award will investigate longitudinal natural history and neurobehavorial assessments, biomarkers, outcome measures, and neurophysiological and neuroimaging correlates of disease severity in Rett syndrome, MECP2 Duplication, CDKL5, and FOXG1 related disorders. Rettsyndrome.org will take a central role in this grant as an administrative partner of the cooperative agreement. THANK YOU to our Rett community who wrote letters and spoke with your legislators. You made a difference in pushing this issue through. What an important announcement to share during October Awareness Month. This very real foundational step forward is a result of successful federal, researcher and private foundation partnership. We look forward to sharing enrollment and participation details in the months to come. #Rettsyndrome #CDKL5 #FOXG1 #MECP2Duplication #ORSAM
Posted on: Wed, 08 Oct 2014 20:42:04 +0000
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