This study is a randomized, double-blind, placebo-controlled study that will evaluate the safety and efficacy of dasiglucagon in children between the ages of 7 days and 12 months who have congenital hyperinsulinism.

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)

The purpose of this study is to look at the safety and tolerability (how well you will react) of the study drug (HM15136) and to determine if it is effective for the treatment of Congenital Hyperinsulinism (HI).

The Panzyga Study is looking at a potential medicine (experimental drug) to see if it can result in increasing platelet counts and may potentially treat ITP.

Phase 1 open-label study to estimate the safety, manufacturing feasibility, and efficacy of intravenously administered, lentivirally transduced T cells expressing anti-CD123 in pediatric subjects with relapsed/refractory Acute Myeloid Leukemia.

This Shifting Perspectives study explores whether we can improve treatment outcomes in teens with anorexia nervosa by adding Adolescent Focused Cognitive Remediation Therapy (CRT) to Family Based Treatment (FBT). CRT focuses on improving cognitive and behavioral flexibility in adolescents.

The purpose of this study is to see if a new investigational drug (Upadacitinib) is safe and well tolerated and also to determine the appropriate dose for subjects with polyarticular course juvenile arthritis (pcJIA).

Our team at the Congenital Hyperinsulinism Center at CHOP is working on a clinical drug trial that involves giving you a study drug called UX033.