Included in this section:|
Acamprosate Fragile X Syndrome Youth Study: The purpose of this study is to learn more about the effectiveness of acamprosate versus placebo as a drug treatment for interfering symptoms associated with fragile X syndrome, such as irritability, hyperactivity and social impairment.
Efficacy, Safety and Tolerability of RO5186582 in Adults and Adolescents with Down Syndrome: A multi-center, randomized, double-blind, placebo-controlled study of the efficacy, safety and tolerability of RO5186582 in adults and adolescents with Down syndrome.
Expressive Language Sampling as an Outcome Measure: The goal of this research study is to learn more about how samples of spoken language can be used to measure change over time in the spoken language, problem solving and behavior of individuals with genetic syndromes like fragile X syndrome.
Fragile X Study: Long-term Safety and Efficacy of Investigational Drug (Adolescent): The purpose of this study is to find out whether an experimental drug is safe and effective for treating fragile X syndrome symptoms.
Fragile X Study: Premutation Carriers: Researchers are investigating neurological symptoms in male and female carriers of the FXS premutation, and comparing these symptoms to other carriers and noncarriers of the same age.
Fragile X Syndrome Treatment With NNZ-2566 Compared to Placebo: The purpose of this study is to evaluate the safety of an oral administration of NNZ-2566 in adolescent and adult males and to determine its drug properties, such as how it is absorbed and broken down in the body.
Nonsense Mutation Dystrophinopathy and Ataluren Treatment Study: Rush University Medical Center is recruiting for the Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in patients with nonsense mutation dystrophinopathy.