Joana Carvalho, PhD,  managing science editor—

Ovid Therapeutics is expecting to have data from a Phase 2 trial testing OV101 (gaboxadol) in teenagers and young adults with fragile X syndrome later this year. The main goal of the randomized double-blind ROCKET study (NCT03697161) is to assess the safety and tolerability of OV101…

A new animal model of fragile X syndrome suggests that a short time window very early in life is critical for effectively treating the condition. The study, “A DNAzyme based knockdown model for Fragile-X syndrome in zebrafish reveals a critical window for therapeutic intervention,” was published in the…

Genetic screening for fragile X syndrome should be available to all pregnant women and to those considering having a child, regardless of family history or fertility issues, a study suggests. The study, “Fragile X syndrome carrier screening accompanied by genetic consultation has clinical utility in populations beyond those…

Derivatives of an Alzheimer’s disease precursor may be valuable biomarkers of disease activity in fragile X syndrome, according to a recent study. The results of the study, “Peripheral Amyloid Precursor Protein Derivative Expression in Fragile X Syndrome,” were published in Frontiers in Integrative Neuroscience. Fragile…

Most caregivers claim that children with fragile X syndrome (FXS) show signs of social avoidance, unresponsiveness or lethargy and irritability, confirming the utility of a set of subscales traditionally used to evaluate the behavior of individuals living with the disease to assess core behavioral symptoms of FXS in…

Scientists have discovered a signaling cascade in fruit flies that may be a promising therapeutic target for treating fragile X syndrome in humans. The study with that finding, “Wnd/DLK Is a Critical Target of FMRP Responsible for Neurodevelopmental and Behavior Defects in the Drosophila Model of Fragile X Syndrome,”…

The presence of AGG triplets in the FMR1 gene sequence may be linked to the number of CGG repeats — the genetic cause underlying fragile X syndrome — and the degree of consanguinity among individuals from the general population, according to a study. The results of the study, “The…

The clinical profile of FMR1 premutation carriers — individuals who have 55 to 200 CGG repeats in their fragile X mental retardation 1 gene sequence instead of the normal 24 to 40 — is markedly different from that of healthy people, a study found. Individuals with that profile…

Posture impairment in young people carrying mutations associated with fragile X-associated tremor/ataxia syndrome may already be present before the onset of the first symptoms of the disorder, a study has found. The study, “Complexity based measures of postural stability provide novel evidence of functional decline in fragile X…

OV101 (gaboxadol) shows an ability to reverse the hyperactivity, irritability, anxiety, aggression and repetitive behaviors that are characteristic of fragile X syndrome, a study in a mouse model of the disease reports. These findings, “Gaboxadol Normalizes Behavioral Abnormalities in a Mouse Model of Fragile X Syndrome,”…