News

U-Lace, FRAXA Partner to Help Fund Research for Fragile X Syndrome

During Autism Awareness Month in April, the nonprofit FRAXA Research Foundation and shoelace company U-Lace established a new partnership to help fund research for the treatment and cure of fragile X syndrome, the most common inherited cause of autism. The partnership will begin with special marketing programs and the development of custom,…

FDA Grants Orphan Drug Status to Investigational Therapy BPN14770 for Fragile X Syndrome

Investigational therapy BPN14770 has received orphan drug status from the U.S. Food and Drug Administration as a treatment for fragile X syndrome, Tetra Discovery Partners recently announced. BPN14770, a small molecule therapeutic agent developed by Tetra Discovery, inhibits the phosphodiesterase type-4D (PDE4D) enzyme linked to memory formation and learning…

NIH Agency Pioneers Collaborative Research into Rare Diseases

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. The Office of Rare Diseases Research (ORDR), headquartered in Bethesda, Maryland, was established in 1993 within the NIH Office of the…

FDA Grants Fast Track Designation to OV101 for Fragile X

The U.S. Food and Drug Administration (FDA) recently granted fast track designation to Ovid Therapeutics’ Fragile X syndrome (FXS) treatment candidate OV101 (gaboxadol). The designation accelerates the development and regulatory review of investigational medications for the treatment of serious conditions and that fill an unmet medical need.

FDA Decision Clears Way for Phase 2 Clinical Trial of BPN14770

The U.S. Food and Drug Administration (FDA) recently accepted the investigational new drug (IND) application for BPN14770, a small molecule therapeutic agent developed by Tetra Discovery Partners for the treatment of Fragile X syndrome and other autism spectrum disorders. Tetra Discovery now plans to launch a Phase 2 clinical…