outcome measures

Outcome measures are how scientists decide whether a treatment works.

Neuren’s Tofinetide Successful in Phase 2 Clinical Trial in Fragile X
In this initial trial with a relatively short treatment period, trofinetide was very well tolerated, with the high dose (70 mg/kg twice daily) demonstrating a consistent pattern of clinical improvement.
Fragile X Cure One Step Closer with FRAXA Support of $1 Million in New Research
4 Countries – 10 Teams – $1 Million for finding treatment targets, pinpointing outcome measures for clinical trials, and attempting to reactivate the gene which is silenced in Fragile X syndrome.
Meltdown no more? Targeting Hypersensitivity in Fragile X
Meet Khaleel Razak, PhD, and Jonathan W. Lovelace, PhD, FRAXA-funded researchers at University of California, Riverside who are tackling Fragile X.
NIH Investigator Carolyn Beebe Smith, PhD, Looks to Improve Sleep in Fragile X Syndrome
Carolyn Beebe Smith studies sleep disruptions in Fragile X and tests whether improving sleep with existing drugs can reduce symptoms and enhance behavior.
Trial and No Error: Better Outcomes for Clinical Trials in Fragile X Syndrome
The team investigated a potential microRNA blood-based biomarker that can be used as a clinical outcome measure for Fragile X syndrome.