Leukodystrophy Center of Excellence News & Announcements

This week’s roundup features research exploring the value of well care as voiced by Black adolescent males and their parents. This and more In The News.

A JAK inhibitor helped patients with Aicardi-Goutières Syndrome meet important developmental milestones.

New insights into Aicardi-Goutières syndrome propel experts to establish qualified outcome measures for clinical drugs.

One year after Drew's leukodystrophy diagnosis at CHOP, the Norton family is having an "amazing run."

The Research Institute's commitment to cultivating and producing strong scientific research is grounded in the diverse mix of experiences, talents, and perspectives that our researchers contribute to our success.

"Diversity has long been a key driver of…

A special space is open on the walls of Adeline Vanderver, MD's, new office for a framed collage of "Commander" Massimo "Mo" Damiani, a toddler wearing an orange jumpsuit who looks ready to join a NASA astronaut team. Dr. Vanderver, a child neurologist…

This week’s stories span discovery (of a potential treatment for rare blood diseases), inspiration (from a mother who transformed grief over her daughter’s terminal disease into a foundation helping other families), and finding practical clinical insight (to distinguish a…