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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
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
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