Rare Disease
- Amyloidosis
- Aromatic L-Amino Acid Decarboxylase Deficiency
- Chronic Immune Thrombocytopenic Purpura
- Chronic Inflammatory Demyelinating Polyneuropathy
- Cystinosis
- Duchenne, Becker and Limb-Girdle Muscular Dystrophy
- Eosinophilic Esophagitis
- Eosinophilic Granulomatosis with Polyangiitis
- Fabry Disease
- Homozygous Familial Hypercholesterolemia
- Lysosomal Acid Lipase Deficiency
- Metachromatic Leukodystrophy
- Neonatal-Onset Multisystem Inflammatory Disease (NOMID)
- Neuromyelitis Optica Spectrum Disorder
- Niemann-Pick Disease Type C
- Pompe Disease
- Primary Hyperoxaluria Type 1
- Spinal Muscular Atrophy
- Sporadic Inclusion Body Myositis
- Von Willebrand Disease