Multiple Sulfatase Deficiency | CHOP Research Institute
 

Multiple Sulfatase Deficiency

Cornerstone Blog

Excellence Award, Orphan Drug Designation, Imaging Technique, Coordinated Asthma Care

Published on
Nov 22, 2024
Excellence Award, Rare Disease, Orthopedic Ultrasound
Congratulations to physician scientist Dr. Yael Mosse, who received a Penn Medicine Award of Excellence for her contributions to pediatric oncology, and more.
Cornerstone Blog

Advances in Gene Therapy, Novel Nanopore Technology, Concussion Science

Published on
Sep 27, 2024
Advances in Gene Therapy, Novel Nanopore Technology
Symposium highlights AAV gene therapy best practices for children, CHOP researchers develop novel nanopore platform, and more In the News

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.

Cornerstone Blog

Making Way for Gene Therapy in Rare Disease

Published on
Aug 31, 2023
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.
Cornerstone Blog

ASCGT 26th Annual Meeting Well-Attended by CHOP Scientists

Published on
May 18, 2023
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.