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Rare Diseases
Topics
- Anthrax
- Ataxia Telangiectasia
- Childhood Leukemia
- Congenital Muscular Dystrophy
- Cystic Fibrosis
- Esophageal Cancer
- Liver Cancer
- Malaria
- Mucopolysaccharidoses (MPS)
- Myasthenia Gravis
- Orphan Drugs
- Osteogenesis Imperfecta
- Pancreatic Cancer
- Spinal Muscular Atrophy
- Usher Syndrome
- Amyotrophic Lateral Sclerosis (ALS)
- Cooley's Anemia
- Hepatitis - A
- Muscular Dystrophy
- Pediatric Cardiomyopathy
- Primary Immunodeficiencies
- Transmissible Spongiform Encephalopathy (TSE)
- Tuberculosis
- Tuberculosis Vaccine
- Hodgkin's Disease
- Orthopox Viruses
- Paget's Disease
- Pediatric Cancer
- Spina Bifida
- Stomach Cancer
- Charcot-Marie-Tooth Disease
- Ovarian Cancer
- Sickle Cell Disease
- Dystonia
- Sudden Infant Death Syndrome
- Valley Fever
- Neuroblastoma
- West Nile Virus
- Neurofibromatosis
- Batten Disease
- Brain Cancer
- Vaginal Cancer
- Fragile X
- Huntington's Disease
- Hydrocephalus
- Rett Syndrome
- Tuberous Sclerosis
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Shaping Therapies for Sickle Cell Disease
Swee Lay Thein is hunting for new ways to help patients with deformed red blood cells.
Cellular Trash-Talking
Rosa Puertollano dives into the mechanisms of cellular cleanup.
One Gene, Three Disorders, and No Cures
Karen Usdin investigates how multiple copies of a short DNA repeat give rise to the Fragile X-related disorders.
The Broken Leg Mystery
Michael Collins and his Clinical Center colleagues cure a teenager of a debilitating bone disorder.
From the Mountains to the Clinic
Sonja M. Best hunts for viruses that evade the immune system.
Molecular Interventions
Christina Annunziata brings her unique expertise to bear on the problem of ovarian cancer.