Emerging autism and Fragile X syndrome treatments

Probiotics and/or microbiota transfer therapies (which improve autism linked gut dysbiosis) and multiple anti-inflammatory drugs are promising therapies for the core symptoms of autism. 

Phosphodiesterase 4D inhibitors restore cyclic adenosine monophosphate (cAMP) levels and increase the quality of life for Fragile X syndrome (FXS) patients.

Bumetanide (which corrects atypical intracellular chloride levels in the brain) and drugs that target the endocannabinoid system may be effective treatments for both autism and FXS.

Despite their promise, poor translatability between preclinical to clinical and Phase 1/2 to Phase 3 has been observed for other potential pharmacotherapies in autism and FXS.

Patient heterogeneity (age, sex, etiology, among others) and lack of biomarkers make it difficult to identify effective treatments in autism and FXS.

https://www.cell.com/trends/pharmacological-sciences/fulltext/S0165-6147(25)00024-0

https://sciencemission.com/autism-and-Fragile-X-syndrome-treatments