Metabolic Disease Program News and Updates

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NIH Awards Penn Medicine and Children’s Hospital of Philadelphia $26 Million Grant to Develop Therapies for Rare Newborn Genetic Diseases

Published on Aug 01, 2023

A Penn Medicine and CHOP team will seek to develop treatments for three rare, incurable genetic diseases with the help of a $26 million grant from the National Institutes of Health.

CHOP Researchers Selected for Bespoke Gene Therapy Consortium

Published on May 24, 2023

CHOP researchers will develop gene therapies for multiple sulfatase deficiency, a devastating lysosomal storage disorder, with the goal of first-in-human trials.

CHOP Expert in Metabolic Diseases Appointed to State Rare Diseases Council

Published on Dec 04, 2017

Can Ficicioglu, MD, PhD, a physician-scientist with expertise in genetic disorders, was appointed to a statewide Rare Diseases Advisory Council by the PA Department of Health.