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Tetra Therapeutics’ investigational oral therapy BPN14770 safely and effectively improves cognitive function, particularly language-related domains, and daily functioning in men with fragile X syndrome, according to data from a Phase 2 clinical trial. “These results offer hope for patients with fragile X syndrome and their families,” Elizabeth…
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Abnormal protein production associated with fragile X syndrome was coupled with altered cell growth that favored fast-growing cell types over those that become nerve cells during early development, a study has shown. Blocking a signaling pathway known as PI3K corrected both excess protein production and…
Better access to information about fragile X-associated ataxia/tremor syndrome (FXTAS) could help improve the quality of life for men carrying fragile X syndrome premutations, according to the results of a small survey. The study, “Men with an FMR1 premutation and their health education needs,” was published in…
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