Outcome measures are how scientists decide whether a treatment works.
Related Articles:
- Trial and No Error: Better Outcomes for Clinical Trials in Fragile X SyndromeJohns Hopkins researcher Christina Timmerman, PhD, searches for a less subjective method to determine if a drug is working in patients with Fragile X syndrome. Many parents of children with Fragile X syndrome were crushed when promising drug trials were unexpectedly stopped a few years ago because subjective behavior-based outcome measures did not justify continuing the trials. The strong feelings linger today. If all goes well with Christina Timmerman’s research, future drug trials may be able to continue with additional metrics for assessment, until there are advanced treatments or even a cure for Fragile X syndrome.
- Fragile X Clinical Trial of AZD7325 in AdultsWith a $51,000 grant from FRAXA Research Foundation, Dr. Craig Erickson conducting a double-blind, placebo-controlled clinical trial of AZD7325 in adults ages 18-50 with Fragile X syndrome at Cincinnati Children's Hospital. The compound being studied is an investigational new drug from AstraZeneca that targets GABA (A) receptors.
- Social Behavior as an Outcome Measure for Fragile X Clinical TrialsOne of the features of the Fragile X mouse model which is relevant to the human Fragile X syndrome (and autism) is social behavior. Several tests show consistent social behavioral abnormalities in the Fragile X mouse model. With a $140,000 grant from FRAXA Research Foundation in 2012-2013, Dr. Willemsen at Erasmus University used social behavior tests to measure the effectiveness of several drug strategies.
- In Their Own Words: Reports From the International Fragile X WorkshopThe 18th International Fragile X and Related Neurodevelopmental Disorders Workshop in Quebec, Canada, was a great success, featuring Fragile X much more heavily than any previous meeting in this series! We asked our speakers to summarize their work in their own words, with brief updates from researchers investigating Fragile X.
- Combinatorial Drug Treatment in a Model of Fragile X Syndrome using Novel BiomarkersWith a $90,000 grant from FRAXA Research Foundation awarded over 2016-2017, University of California researchers Khaleel Razak, PhD, and Jonathan W. Lovelace, PhD, are exploring drug combinations to limit hypersensitivity to sounds in Fragile X mice.