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CENTERS OF EXCELLENCE
Cell and Gene Therapy
Acid Sphingomyelinase Deficiency
Alcohol Dependence
Alzheimer's Disease
Atypical Hemolytic Uremic Syndrome
Cartilage Disorders
CDKL5 Deficiency Disorder
Ceroid lipofuscinosis 2
Complex Partial Seizures
Cryopyrin-Associated Periodic Syndromes (CAPS)
Cystinuria
Deficiency of Interleukin-1 Receptor Antagonist (DIRA)
Duchenne Muscular Dystrophy
Dystrophic Epidermolysis Bullosa (DEB)
Fabry Disease
Gaucher Disease Type 1
Growth Hormone Deficiency
Hemophilia
Hereditary Angioedema
Hereditary ATTR amyloidosis (hATTR)
Homozygous Familial Hypercholesterolemia (HoFH)
Idiopathic Pulmonary Fibrosis
Keratoconus
Long-Chain Fatty Acid Oxidation Disorders
Migraine
Mucopolysaccharidosis
Opioid Dependence
Paroxysmal Nocturnal Hemoglobinuria
Pompe Disease
Primary Hyperoxaluria Type 1
Recurrent C. difficile Infection
Recurrent Pericarditis
Schizophrenia
Spinal Muscular Atrophy
Tardive Dyskinesia
Tumor-induced Osteomalacia
Type 1 Diabetes
Venous Thromboembolism
X-linked Hypophosphatemia
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Tommy (DMD)
Sienna and Wesley (SMA)
Erin (XLH)
Nicholas (MPS)
Robert (IPF)
Amy (HAE)
Joan (TD)
Wanda (TD)
Annabelle (MPS)
Austin & Max (DMD)
Lisa (HAE)
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