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Research Study for Individuals with Hyperinsulinism, Type 1 Diabetes Mellitus, and Healthy Children
Our team is working on a study to better understand neurological problems, including seizures and developmental delays, that occur in children with hyperinsulinism, including HI/HA syndrome, and type 1 diabetes mellitus.
Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism (for children under 1 year of age)
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.
Dova Study for Children with Immune Thrombocytopenia (ITP)
The Dova study is for children below 18 years with primary Immune Thrombocytopenia (ITP) who have had an insufficient response to previous treatment.
DTX401 Gene Therapy in Glycogen Storage Disease (GSD) Type Ia
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)
HM15136 for the Treatment of Congenital Hyperinsulinism Research Study
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).
Phenotypes of Congenital Hyperinsulinism
We are doing this study to see if the patterns of abnormalities in controlling insulin in children and adults with hyperinsulinism are related to any underlying genetic cause and to identify possible new genetic causes.
Phenotyping Congenital Hyperinsulinism
The Congenital Hyperinsulinism Center at the Children's Hospital of Philadelphia is working on a research study to better understand how people with hyperinsulinism may have different blood sugar responses to certain tests (like fasting or drinking a high-protein shake) when compared to people without hyperinsulinism.
The Myelin Disorders Biorepository Project (MDBP)
This study involves the collection of medical information and samples from people with a confirmed or suspected leukodystrophy or related disorder affecting the white matter of the brain, as well as "healthy controls" who are not known to be affected by one of these disorders.
UX053 in GSD III
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.