Mary Chapman,  features writer—

The Ultimate Fighting Championship (UFC) lifted the spirits of a boy named Sebastian recently while helping to make everyone more aware of Batten disease, the foe Sebastian and his family are fighting. Earlier this year, Sebastian’s father, Chris Velona, asked UFC to help raise awareness of Batten, a genetic disorder…

Nationwide Children’s Hospital, the site of a first-of-its-kind clinical trial in Batten disease, has dedicated its lobby in honor of the Charlotte and Gwenyth Gray Foundation to Cure Batten Disease. Touted as one of the world’s largest and most comprehensive pediatric academic medical centers, the hospital is in…

To advance its commitment to research aimed at improving the lives of those with rare metabolic disorders such as Batten disease, Amicus Therapeutics is establishing a Global Research and Gene Therapy Center of Excellence in Philadelphia, Pennsylvania. The 75,000-square-foot center, scheduled to open this year, will be in…

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 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…

Kennedy Hansen Kennedy Hansen had already lost her vision and most of her motor skills to Batten disease by 2013, but when the head of her cheerleader squad went around a circle at practice one day asking each member to name their life…