Multiple Sulfatase Deficiency | CHOP Research Institute
 

Multiple Sulfatase Deficiency

Gene Therapy for Inherited Metabolic Disorders Program

Gene Therapy for Inherited Metabolic Disorders Program

Providing cutting-edge molecular therapies for patients with inborn errors of metabolism in a first of its kind, word-class clinic.

Making Way for Gene Therapy in Rare Disease

Published on
Aug 31, 2023
in Cornerstone Blog
Laura Adang. MD, PhD, (left) and Rebecca Ahrens-Nicklas, MD, PhD, holding one of their dear patients
Children’s Hospital of Philadelphia is the lead study site for the development of a gene therapy for multiple sulfatase deficiency.

ASCGT 26th Annual Meeting Well-Attended by CHOP Scientists

Published on
May 18, 2023
in Cornerstone Blog
ASCGT 26th Annual Meeting Well-Attended by CHOP Scientists
Scientists from Children’s Hospital of Philadelphia attend the American Society of Cell & Gene Therapy Annual Meeting to share their expertise through posters, panel discussions, and workshops.

Leukodystrophy Center of Excellence

Leukodystrophy Center of Excellence

Using an integrated and multidisciplinary approach to addressing the unique needs of infants, children and adolescents with inherited white matter diseases.