Rare Disease

• Advanced Systemic Mastocytosis

• Alpha-1 Antitrypsin Deficiency

• Barth Syndrome

• Beta Thalassemia

• Chronic Inflammatory Demyelinating Polyradiculoneuropathy

• Distal Renal Tubular Acidosis

• Dravet Syndrome

• Eosinophilic Gastroenteritis

• Esophageal or Gastroesophageal Junction Cancer (EC/GEJC)

• Gaucher Disease

• Glut-1 Deficiency Syndrome

• Hemophilia

• Mucopolysaccharidosis I & II

• Multifocal Motor Neuropathy

• Multiple Myeloma

• Multiple Sclerosis

• Muscular Dystrophy

• Narcolepsy

• Niemann-Pick Disease Type C

• Polycythemia Vera

• Primary Hyperoxaluria

• Sickle Cell Disease

• Thrombocytopenia

• Thrombotic Thrombocytopenic Purpura

• X-linked Hypophosphatemia (XLH)