Table 1 Classification of Diseases included in rare disease clinical trials from 2013 to 2022
From: Trend of clinical trials of new drugs for rare diseases in China in recent 10 years
Category | Disease |
|---|---|
Blood system | Hemophilia |
Hypercholesterolemia | |
Metabolic system | Hypercholesterolemia |
Gaucher’s Disease | |
Hyperphenylalaninemia | |
Hypophosphatemia Rickets | |
Fabry Disease | |
Mucopolysaccharidosis | |
Respiratory system | Idiopathic Pulmonary Fibrosis |
Neuroscience | Neuromyelitis Optica |
Severe myoclonic Epilepsy in Infancy (Dravet syndrome) | |
Parkinson Disease | |
Generalized Myasthenia Gravis | |
Spinal Muscular Atrophy | |
Amyotrophic Lateral Sclerosis | |
Huntington Disease | |
Charcot-Marie-Tooth Disease | |
Autoimmune system | Paroxysmal Nocturnal Hemoglobinuria |
Autoimmune Encephalitis | |
Multiple Sclerosis | |
Hereditary disorders | Charcot-Marie-Tooth Disease |
Hereditary Angioedema (HAE) | |
Spinocerebellar Ataxia | |
Histiocytic diseases | Langerhans Cell Histiocytosis |