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The purpose of this study is to determine the safety and tolerability of an experimental form of therapy, known as UCART22 ("study drug").

The goal of this research study is to compare sex and gender development differences between children ages 4-8 years who are on the autism spectrum with children who are not on the autism spectrum.

We want to learn more about how the brains of children on the autism spectrum process sounds and language.

This study is being done to understand how the brain of autistic adolescents and adults process sound and language.  We hope the information gained from this study may lead to future therapies for language difficulties in autistic individuals.

Evaluate the safety and efficacy of an investigational drug called "Elamipretide" in adult patients with mitochondrial disease.

Study focusing on non-invasive MRI procedures to measure biological activity within leg muscles of individuals, including both healthy volunteers and those with mitochondrial disease.

We are recruiting patients with genetically confirmed primary mitochondrial myopathy who are at least 18 years old for a randomized, double-blind, placebo-controlled study of a new drug to treat this disorder.

Our team at the Congenital Hyperinsulinism Center at CHOP is working on a research study to determine whether an experimental gene therapy product, DTX401, is safe and effective in people with glycogen storage disease type Ia (GSDIa)

Transcend, Triumph, and Thrive (3T) is a HIV prevention intervention study for transgender youth ages 15-24, looking at their use of Pre-exposure prophylaxis (PrEP). The purpose of this study is to pilot a program designed to address the health needs of transgender youth.

Patients with Krabbe disease do not have enough of a certain protein called galactosylceramidase due to a defective gene. The lack of this protein causes damage to the patient's nerve cells. In this study, we will give a normal, properly working copy of that gene to your child.