Ana de Barros, PhD, managing science editor —

Two 5-year-olds participating in a trial evaluating the first treatment for Batten disease were reunited at Le Bonheur Children’s Hospital in Memphis after Hurricane Harvey almost suspended the treatment for one of them. Elle Gieselmann and Micah McCorkle are among the few Americans receiving the enzyme replacement therapy. The…

The U.S. Food and Drug Administration (FDA) granted orphan drug status to Polaryx Therapeutics’ PLX-200 in response to a lack of treatments available for late infantile neuronal ceroid lipofuscinosis (LINCL), a type of Batten disease. PLX-200 is a repurposed drug (one that was developed for one indication…

A small North Carolina nonprofit organization, Indo Jax Surf Charities, has recently gained attention for its set of free programs to help children with Batten disease and other special needs build self-esteem. Since launching these programs, Indo Jax has been featured in several media platforms, including American Way…

The Batten Disease Support and Research Association (BDSRA) will hold its 2017 Family Conference in July, bringing together patients and families touched by Batten disease. The on July 6–9 event will take place at the Wyndham Grand Pittsburgh Downtown. The conference joins family members, children and researchers to…