Newborn screening for lysosomal storage disorders and other neuronopathic conditions.Matern D, Oglesbee D, Tortorelli S.; Dev Disabil Res Rev. 2013 Jun;17(3):247-253. doi: 10.1002/ddrr.1117.
Keywords: Newborn screening (NBS), public health program, 13 lysosomal storage disorders, X-adrenoleukodystrophy, Wilson disease, Friedreich ataxia.
Wednesday, June 26, 2013
Final Study Record Detail: Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia
Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia. www.clinicaltrials.gov, A service of the U.S. National Institutes of Health.
Study Design:
Allocation: Randomized; Endpoint Classification: Efficacy Study; Intervention Model: Parallel Assignment; Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor); Primary Purpose: Supportive Care
Due to small sample size and considering there are no specific studies in this population with EGb761; calculation with the use of a statistical hypothesis was not possible. Primary efficacy analyses performed on the mITT population and analysis of safety performed on the safety population.
Study Design:
Allocation: Randomized; Endpoint Classification: Efficacy Study; Intervention Model: Parallel Assignment; Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor); Primary Purpose: Supportive Care
Due to small sample size and considering there are no specific studies in this population with EGb761; calculation with the use of a statistical hypothesis was not possible. Primary efficacy analyses performed on the mITT population and analysis of safety performed on the safety population.
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