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CTX is typically a highly progressive disease, with case reports of symptoms that first appear at any time from birth through adulthood and then worsen over time.
Cure Glomerulonephropathy Network (CureGN) is a multi-year study of 2,400 children and adults with four rare glomerular (kidney) diseases at over 50 centers in the US, Canada, and Italy.
CHOP, in collaboration with Janssen Research & Development, is conducting a study for children and young adults up to 30 years old, with relapsed or refractory precursor T-cell acute lymphoblastic leukemia or lymphoblastic lymphoma.
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.
We are studying an investigational drug called sodium dichloroacetate (DCA).
The purpose of this study is to find out how well surveys can measure disease activity in kids with lower back arthritis. The study will last for either 3 or 6 months and take 2 or 3 visits.
The purpose of this study is to see if two biomarkers, called CP and PCT, can help physicians identify when patients have an infection versus a flare of their sJIA.
The Dova study is for children below 18 years with primary Immune Thrombocytopenia (ITP) who have had an insufficient response to previous treatment.
Our Driving after Concussion study needs the help of both healthy and recently concussed teen partners in our research to understand how concussion affects driving behaviors in teens.