Mary Chapman, features writer —

In the documentary “Minds Wide Open,” which spotlights advances in brain research, Monica Coenraad is cautiously hopeful about her daughter Chelsea’s future with Rett syndrome. “I don’t expect a 100 percent ‘like it never ever even happened’ cure for Chelsea, but I do think that there can be improvements,”…

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…

The Rett Syndrome Research Trust (RSRT) has awarded $10 million to a host of top international researchers to help find a cure for the neurological disorder. The 2018 award is the organization’s largest since its founding in 2008. To date, RSRT has awarded some $57 million for research. The…

The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how…