Table 3 Diseases associated with at least five RPD designated products (n = 26)
Most designated diseases | Number of RPD designations | Percentage of total RPD designations | Therapeutic area |
|---|---|---|---|
Duchenne muscular dystrophy | 30 | 5 | Neurology |
Neuroblastoma | 21 | 4 | Oncology |
Sickle cell disease | 18 | 3 | Hematology |
Diffuse intrinsic pontine glioma* | 16 | 3 | Oncology |
Osteosarcoma | 15 | 3 | Oncology |
Acute lymphoblastic leukemia | 10 | 2 | Oncology |
Cystic fibrosis | 8 | 1 | Pulmonary |
Epidermolysis bullosa | 8 | 1 | Dermatology |
Congenital isolated hyperinsulinism | 7 | 1 | Endocrinology |
Dravet syndrome | 7 | 1 | Neurology |
Ewing sarcoma | 7 | 1 | Oncology |
Friedreich ataxia | 7 | 1 | Neurology |
GM2 gangliosidosis# | 7 | 1 | Metabolism |
B-thalassemia | 6 | 1 | Hematology |
Medulloblastoma | 6 | 1 | Oncology |
Mucopolysaccharidosis type 1 | 6 | 1 | Metabolism |
Propionic acidemia | 6 | 1 | Metabolism |
Spinal muscular atrophy | 6 | 1 | Neurology |
Angelman syndrome | 5 | 1 | Neurology |
Gaucher disease | 5 | 1 | Metabolism |
Krabbe disease | 5 | 1 | Neurology |
Lennox-Gastaut syndrome | 5 | 1 | Neurology |
Mucopolysaccharidosis type 2 | 5 | 1 | Metabolism |
Netherton syndrome | 5 | 1 | Dermatology |
Rett syndrome | 5 | 1 | Neurology |
Rhabdomyosarcoma | 5 | 1 | Oncology |