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A CTSU snapshot

A sampling of studies currently underway at the unit

Phenotypic and genetic factors in autism spectrum disorders
Louis Kunkel, PhD, Director, Program in Genomics, Children's Hospital Boston

Measuring disease progression in spinal muscular atrophy with electrical impedance myography
Basil Darras, MD, Director, Neuromuscular Program

Xolair and oral tolerance induction in milk-allergic children
Dale Umetsu, MD, PhD, Division of Allergy/Immunology

A randomized trial of hormonal add-back therapy in adolescents treated with GNRH agonists for endometriosis
Catherine Gordon, MD, MSc, Director, Bone Health Program

Effects of adrenal and gonadal hormone replacement in young women with anorexia nervosa
Catherine Gordon, MD, MSc, Director, Bone Health Program

A randomized trial of Lovaza in adolescents with mild to moderately elevated triglycerides
Stavroula Osganian, MD, ScD, MPH, Director, Clinical Research Program

Allergens in inner-city schools and childhood asthma
Wanda Phipatanakul, MD, MS, Division of Allergy/Immunology

The efficacy and safety of monthly therapy with recombinant Factor XIII in subjects with congenital Factor XIII deficiency
Ellis Neufeld, MD, PhD, Associate Chief, Division of Hematology/Oncology

Nutritional treatment of overweight adolescents with cardiovascular risk factors
Sarah de Ferranti, MD, MPH, Director, Preventive Cardiology Clinic

Body composition and intestinal adaptation in pediatric short bowel syndrome
Nilesh Mehta, MD, Pediatric Critical Care Nutrition Program

Upcoming studies in the CTSU

Gene therapy for severe combined immunodeficiency (SCID-X1) using a self-inactivating gammaretroviral vector
Luigi Notarangelo, MD, Division of Allergy/Immunology

A phase II trial of zoledronic acid, pravastatin and lonafarnib for Hutchinson-Gilford Progeria Syndrome and progeroid laminopathies
Mark Kieran, MD, PhD, Director, Pediatric Medical Neuro-Oncology

A dose-escalation tolerability study of levodopa/carbidopa in Angelman syndrome
Wen-hann Tan, MD, Division of Genetics

Health and psychosocial outcomes in adults with classic galactosemia
Gerard Berry, MD, Director, Metabolism Program

Preventing acute chest syndrome in sickle cell disease through transfusion: a feasibility study
Matthew Heeney, MD, Director, Hematology Clinic; Clinical Director, Sickle Cell Program

 

 

 

 

 

 

VIDEO: Children speak: What it's like to be a medical research subject

A day in the life of the CTSU, in pictures

Upcoming CTSU studies

Video series: Follow Brett as his body learns to tolerate milk

CHB-Connect: Volunteer for clinical research

A Parent's Guide to Medical Research

A parent turned study coordinator discusses pediatric research

Making brain imaging child's play

 

 

 

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