Mary Chapman,  features writer—

An Indiana University-affiliated venture fund has invested $200,000 in Confluence Pharmaceuticals to continue development of the company’s lead product for the treatment of fragile X syndrome. Along with AOP Orphan Pharmaceuticals, Confluence is developing a novel, clinically discovered adult and pediatric therapy for social and communication impairments…

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…

A new collaboration between Tetra Discovery Partners and Shionogi & Co. is aimed at accelerating the development of an innovative treatment called BPN14770 for fragile X syndrome and other disorders in strategic Asian markets. Valued at up to $160 million, the deal is expected to enable clinical-stage…

Fragile X syndrome testing will now be available for newborns in North Carolina by way of a new study led by RTI International to research the disease’s prevalence and the benefits of early treatment. Called Early Check, the free-screening study is designed to identify children with rare health…