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Families of SMA has Funded the Following Clinical Research through the Project Cure SMA Trial Network: 1) SMA Natural History and Outcome Measure Development
Including: MUNE Studies, SMA Pregnancy Studies, Natural History Studies in all SMA Types, and Development of the CHOP INTEND Outcome Measure.
With funding of $571,865 to the following sites: University of Utah, Ohio State University, Johns Hopkins University, and The Children's Hospital of the University of PA. 2) Open Label Phase I Study of VPA and PBA.
Including: Assessment of Safety, Tolerability and Effect on SMN Expression of Valproic Acid and Sodium Phenylbutyrate in SMA Patients.
With funding of $633,276 to The University of Utah. 3) Multicenter Phase II CARNIVAL Trial.
Including: Double-Blind, Randomized, Placebo-Controlled Trial of L-Carnitine and Valproic Acid in Spinal Muscular Atrophy Type II and III.
With funding of $1,426,806 to the following sites: University of Utah, University of Wisconsin-Madison Health Sciences, Wayne State University, Ohio State University, Johns Hopkins Medical School, and Ste. Justine Hospital. 4) Phase II VALIANT Trial.
Including: Double-Blind, Randomized, Placebo-Controlled Trial of L-Carnitine and Valproic Acid In Ambulant Adults with SMA.
With funding of $205,059 to The Ohio State University. 5) Multicenter Open Label CARNI-VAL Type I Trial.
Including: Assessment of Safety, Tolerability of L-Carnitine and Valproic Acid In Infants with SMA Type I.
With funding of $1,213,352 to the following sites: University of Utah, University of Wisconsin-Madison Health Sciences, Wayne State University, OhioState University, Johns Hopkins Medical School, Ste. Justine Hospital, and Duke University Medical Center. 6) Central Database Coordinating Center from 2005 to 2010.
Including: Staff to Generate Database, QA and Entry and Analysis of Clinical Trial Data. With funding of $488,285 to The University of Utah. 7) Supporting Functions for the Project Cure SMA Trial Network from 2004 to 2010.
Including: Clinical Trials Manager, Network Statistician, Lead PI for Outcome Measure Development and QA, Central Pharmacy, Medical Safety Monitor, and Laboratory Tests for SMN Copy Number and Expression.
With Funding of $1,675,634 to the following sites: University of Utah, CBS Squared, Inc., Northwestern University, Ohio State University, and University of Manitoba. Total Funding for Clinical Research of $6,435,707.
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