Clinical and Laboratory Study of Methylmalonic Acidemia

Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:

Natural History Study Protocol in PMM2-CDG (CDG-Ia)

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

GM-CSF for Immunomodulation Following Trauma (GIFT) Study

Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC)

Peak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy

Patient Centered Comprehensive Medication Adherence Management System in Patients With Sickle Cell Disease

A Study of Dulaglutide (LY2189265) in Children and Adolescents With Type 2 Diabetes

A Phase 2 Study of Ruxolitinib With Chemotherapy in Children With Acute Lymphoblastic Leukemia

Review of Charts From Amish/Mennonite Variant PA Patients

This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC 1 or 2

Improving Medication Adherence in Adolescents Who Had a Liver Transplant

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

Evaluating the Genetic Causes and Progression of Cholestatic Liver Diseases (LOGIC)

Improving Safety and Quality of Tracheal Intubation Practice in Pediatric ICUs

A Study of Lymphoseek® as a Lymphoid Tissue Targeting Agent in Pediatric Patients With Melanoma, Rhabdomyosarcoma, or Other Solid Tumors Who Are Undergoing Lymph Node Mapping

BIOPIC: Fungal Biomarkers for Diagnosis and Response to Therapy for Pediatric Candidemia

Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)

Study of N91115 in CF Patients Who Are Heterozygous for F508del-CFTR and a Gating Mutation and Being Treated With Ivacaftor

TODAY2 Phase 2 Follow-up

Amish/Mennonite Research Contact Registry

STRIDE Biorepository

Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia

Elbasvir (EBR)/Grazoprevir (GZR) in Pediatric Participants With Chronic Hepatitis C Infection (MK-5172-079)

Dyslipidemia of Obesity Intervention in Teens

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

Efficacy and Safety of Viaskin Milk in Children With IgE-Mediated Cow's Milk Allergy

A Study of Ramucirumab (LY3009806) in Children With Refractory Solid Tumors

Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

Inhaled Sodium Nitrite as an Antimicrobial for Cystic Fibrosis

Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503)

Safety and Efficacy Study of Viaskin Peanut in Peanut-allergic Young Children 1-3 Years of Age

A Phase I Study Of Etrolizumab Followed By Open-Label Extension And Safety Monitoring In Pediatric Patients With Moderate To Severe Ulcerative Colitis Or Moderate To Severe Crohn's Disease

AFQ056 for Language Learning in Children With FXS

Prognostication Biomarkers in Pediatric Cardiac Arrest

GM-CSF for Reversal of immunopAralysis in pediatriC sEpsis-induced MODS Study

PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

Childhood Cancer Survivor Study

Longitudinal Study of Mitochondrial Hepatopathies

A Pilot Study to Evaluate the Hypoglossal Nerve Stimulator in Adolescents With Down Syndrome and Obstructive Sleep Apnea

Serum Ropivacaine Concentrations in Pediatric Patients Receiving Continuous Wound Catheter Analgesia

Trial of Panobinostat in Children With Diffuse Intrinsic Pontine Glioma

FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)

A Multicenter Study to Obtain Retrospective Data for Subjects Previously Diagnosed With Adenovirus Infection to Serve as Matched Historical Controls for Study CMX001-304

Vedolizumab IV in Pediatric Participants With Ulcerative Colitis (UC) or Crohn's Disease (CD)

Randomized Controlled Trial of Valganciclovir for Cytomegalovirus Infected Hearing Impaired Infants

Pediatric Longitudinal Cohort Study of Chronic Pancreatitis

Smith-Lemli-Opitz Syndrome and Cholic Acid

Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults With Blood Cancers Undergoing Donor Stem Cell Transplant

A Study of Intrathecal SHP611 in Participants With Late Infantile Metachromatic Leukodystrophy