Disease

Niemann-Pick Disease, Type C

About the Disease
Niemann-Pick Disease, Type C1, also known as niemann-pick disease, type c, is related to niemann-pick disease type c, juvenile neurologic onset and niemann-pick disease type c, adult neurologic onset, and has symptoms including cerebellar ataxia, muscle spasticity and seizures. An important gene associated with Niemann-Pick Disease, Type C1 is NPC1 (NPC Intracellular Cholesterol Transporter 1), and among its related pathways/superpathways are Neuroscience and "Plasma lipoprotein assembly, remodeling, and clearance". The drugs Miglustat and Anti-Infective Agents have been mentioned in the context of this disorder. Affiliated tissues include eye, bone marrow and liver, and related phenotypes are dysphagia and jaundice

Common Targets
Protein kinase C (nonspecified subtype) | gamma-Secretase | HDAC1 | GABA(A) receptor | DYRK1A | UGCG | HDAC3 | Cholesterol esterase (nonspecified subtype) | S1PR4 | CLK4 | TWNK | Chaperone (nonspecified subtype) | S1PR3 | SLC5A4 | Heat shock protein 70 (nonspecified subtype) | GAA | S1PR5 | CLK1 | APOE | MIR4709 | KRR1 | GBA2 | RIPK1 | S1PR1 | NPC1 | HDAC6 | HDAC2 | NPC2 | SNCA

疾病靶点研报
Niemann-Pick disease, type C

Note: If you'd like to get a target analysis report for Niemann-Pick Disease, Type C, or if you are interested to learn how our AI-powered BDE-Chem can design therapeutic molecules to interact with the target(s) above against the disease of Niemann-Pick Disease, Type C at a cost 90% lower than traditional approaches, please feel free to contact us at BD@silexon.tech.

Other Diseases

Charcot-Marie-Tooth Disease Axonal Type 2N | Nephrocalcinosis | Metachondromatosis | Sleep Apnea, Obstructive | Norrie Disease | Marinesco-Sjogren Syndrome | Autosomal Recessive Spastic Paraplegia Type 54 | Multiple Epiphyseal Dysplasia | Neuroma | Traboulsi Syndrome | Waardenburg Syndrome Type 2E | Osteoporosis | Urethritis | Vasculitis | Peeling Skin Syndrome Type B | Blomstrand Osteochondrodysplasia | Bartsocas-Papas Syndrome | Craniometaphyseal Dysplasia | Geleophysic Dysplasia | Erythematotelangiectatic Rosacea | Heroin Dependence | Pachyonychia Congenita | Netherton Syndrome | Primary Lateral Sclerosis | Multiple System Atrophy | Conjunctivitis, Allergic | Binge Eating Disorder | Ameloblastic Carcinoma | Lentigo | Proteasome-associated Autoinflammatory Syndrome 2 | Thyroid Dysgenesis | Myoclonus-dystonia Syndrome | Juvenile Polyposis | Crisponi Syndrome | Oral Lichen Planus | Cutaneous Lupus Erythematosus | Systemic Lupus Erythematosus | Primary Cutaneous Amyloidosis | Larsen Syndrome | Actinomycetoma | Kaposiform Hemangioendothelioma | Macrophage Activation Syndrome | Eiken Syndrome | Eczema | Thyroiditis | Mucolipidosis Type II | Xeroderma Pigmentosum Variant Type | Congenital Primary Aphakia | Carey-Fineman-Ziter Syndrome | Aldosterone Synthase Deficiency | McKusick Type Metaphyseal Chondrodysplasia | LEOPARD Syndrome | Central Retinal Artery Occlusion | Silver-Russell Syndrome | Chorea-acanthocytosis | Bartter Syndrome | Chronic Granulomatous Disease, X-linked | Dystonia Musculorum Deformans | Charcot-Marie-Tooth Disease Type 2T | Diabetes Insipidus, Nephrogenic | Bardet-Biedl Syndrome | Rash | Tardive Dyskinesia | Granuloma Annulare | Bullous Pemphigoid | Corneal Neovascularization | Hypertension | Congenital Stationary Night Blindness | Anorchia | Pneumonia, Bacterial | Inflammatory Myofibroblastic Tumor | Sponastrime Dysplasia | Lateral Meningocele Syndrome | Rhabdomyosarcoma, Embryonal | Beare-Stevenson Syndrome | Autism Spectrum Disorders | Cutaneous T-cell Lymphoma | Asthma, Nocturnal | Sertoli Cell-only Syndrome | Pancreatitis, Chronic | Renal Hypouricemia | Kashin-Beck Disease | Chronic Kidney Disease | Sezary Syndrome | Craniofrontonasal Syndrome | Diverticulitis | Ovarian Sex Cord-stromal Tumor | Autonomic Neuropathy | Waardenburg Syndrome Type 2 | Auriculocondylar Syndrome | Cerebrotendinous Xanthomatosis | Nephronophthisis | Combined Pituitary Hormone Deficiency | Chronic Myelomonocytic Leukemia | Carney Triad | Angiosarcoma Of The Breast | Epidermolysis Bullosa Dystrophica | Polycystic Kidney, Autosomal Dominant | Cherubism | Rolandic Epilepsy | Spondyloepiphyseal Dysplasia Tarda, X-linked | Meniere's Disease | Eccrine Porocarcinoma | POEMS Syndrome | Glaucomatocyclitic Crisis | Klippel-Feil Syndrome | Lipoma | Hairy Cell Leukemia | Tetraplegia | Necrotizing Autoimmune Myopathy | Barakat Syndrome | Hepatitis, Chronic | Language Disorders | Leri-Weill Dyschondrosteosis | Teratozoospermia | Cholestasis, Intrahepatic | Gerodermia Osteodysplastica | Cancer, Lung | Blue Rubber Bleb Nevus Syndrome | Apparent Mineralocorticoid Excess Syndrome | Alveolar Capillary Dysplasia | Brachydactyly | Neovascular Glaucoma | Axenfeld-Rieger Syndrome | Erythropoietic Protoporphyria | Presbycusis | Krabbe Disease | Brugada Syndrome 1 | Disseminated Intravascular Coagulation | Hereditary Sensory Neuropathy Type 1 | Chromosome 9q34.3 Deletion Syndrome | Encephalopathy, Ethylmalonic | Pleural Tuberculosis | Hypoproteinemia, Hypercatabolic | Schwannoma | Huntington's Disease-like 2 | Turner's Syndrome | Corneal Ulcer | Carcinoid Syndrome | Martsolf Syndrome | Conduct Disorder | Pseudoexfoliation Syndrome | Trichotillomania | Hereditary Elliptocytosis | Granular Corneal Dystrophy | Ichthyosis Bullosa Of Siemens | Craniofacial Dysostosis | Primary Progressive Nonfluent Aphasia | Periodontitis | Familial Exudative Vitreoretinopathy | Glycogen Storage Disease Type 1 | Lymphoma, B-cell | Hepatitis, Autoimmune | Scleroderma | Craniopharyngioma | High Molecular Weight Kininogen Deficiency | Cardiac Arrest | Lymphoproliferative Disease, X-linked | Nance-Horan Syndrome | Chronic Thromboembolic Pulmonary Hypertension | Histoplasmosis | Congenital Hemolytic Anemia | Congenital Poikiloderma | Bone Giant Cell Tumor | Amebiasis | Osteogenesis Imperfecta Type IV | Chronic Granulomatous Disease | Dementia, Vascular | Crohn's Disease | Overactive Bladder | Okihiro Syndrome | Lymphoma | Waardenburg Syndrome Type 1 | Hepatitis A | Glycogen Storage Disease Type 9 | Aicardi-Goutieres Syndrome | Open-angle Glaucoma | Bethlem Myopathy | Multiple Congenital Anomalies-hypotonia-seizures Syndrome 2 | Eclampsia | Retinitis Pigmentosa | Majeed Syndrome | Macular Corneal Dystrophy Type 1 | Arthropathy | Chitayat Syndrome | Myelitis, Transverse | Glycogen Storage Disease Type 6 | Congenital Bilateral Absence Of Vas Deferens | Eating Disorder | Hereditary Multiple Exostoses | Constipation | Ameloblastoma | Thalassemia, Beta | Epidermolytic Palmoplantar Keratoderma | Harlequin Ichthyosis | Neurodermatitis | Tyrosinemia Type 2 | Sturge-Weber Syndrome | Angiodysplasia | Psoriasis | Hepatitis C, Chronic | Congenital Fiber-type Disproportion Myopathy | Myotonia | Hemorrhoids | Tricho-hepato-enteric Syndrome | Craniosynostosis | Congenital Heart Defects | PASLI Disease | Cyclic Vomiting Syndrome | REM Sleep Behavior Disorder | Coffin-Lowry Syndrome | Atelosteogenesis Type 2 | Facioscapulohumeral Muscular Dystrophy Type 1 | Stuttering | Pierre Robin Syndrome | Hypermethioninemia | Epidermolysis Bullosa Acquisita | Osteochondrosis | Epilepsy Of Infancy With Migrating Focal Seizures | Pineoblastoma | Optic Neuritis | Gastrointestinal Disorders | Galloway-Mowat Syndrome | Hepatoblastoma | Familial Hyperaldosteronism | Delirium | Sialidosis | Pregnancy, Ectopic | Lyme Disease | Pitt-Hopkins Syndrome | Fuchs Heterochromic Iridocyclitis | Neuroendocrine Cancer | Nager Acrofacial Dysostosis | Dengue Shock Syndrome | Hypercalcemia | Hydrops Fetalis | Rosacea | Porphyria Cutanea Tarda | Osteosarcoma | Spinal Muscular Atrophy Type 2 | Retinal Coloboma | Lafora Disease | Porokeratosis | Epidermodysplasia Verruciformis | Megacystis-microcolon-intestinal Hypoperistalsis Syndrome | Insulin Resistance | Glycogen Storage Disease Type 5 | Phenylketonuria | Myelitis | Heterotopic Ossification | Cutaneous Mastocytosis | Waardenburg Syndrome | Oligodendroglioma | B-cell Prolymphocytic Leukemia | Cutaneous Angiosarcoma | Familial Hemiplegic Migraine | Imerslund-Grasbeck Syndrome | Dysplastic Nevus | Spitzoid Melanoma | Myoclonic Epilepsy With Ragged Red Fibers | Malnutrition | Absence Epilepsy | Arteriovenous Malformations | Tinea | Lymphoproliferative Disorders | Acute Tubular Necrosis | Seizures | Cutis Laxa | C3 Glomerulopathy | Uveitis | Oculocutaneous Albinism | T-cell Prolymphocytic Leukemia | Charcot-Marie-Tooth Disease | Poretti-Boltshauser Syndrome | Pseudomyxoma Peritonei | Familial Retinal Arterial Macroaneurysm | Central Core Disease | Kabuki Syndrome 2 | Double Outlet Right Ventricle | Rickets | KBG Syndrome | Ischemia | Endophthalmitis | Seizures-scoliosis-macrocephaly Syndrome | Mycosis Fungoides | Wiskott-Aldrich Syndrome | Epidermolytic Ichthyosis, Annular | Carcinoma, Squamous Cell | VACTERL Association | Spinocerebellar Ataxia Type 20 | Peeling Skin Syndrome, Acral Type | Hemorrhagic Disorders | Leukemia | Craniolenticulosutural Dysplasia | Intestinal Tuberculosis | Fukuyama Congenital Muscular Dystrophy | Carcinoid Tumor | Glycogen Storage Disease | Nemaline Myopathy 10 | Esophagitis | Autosomal Recessive Bestrophinopathy | Greig Cephalopolysyndactyly Syndrome | Congenital Afibrinogenemia | Distal Spinal Muscular Atrophy | Cellulitis | Autosomal Recessive Spastic Paraplegia Type 75 | Bleeding Disorder Due To CalDAG-GEFI Deficiency | Arthritis, Reactive | Nevus | Congenital Aniridia | Fibrosis | Bruck Syndrome | Focal Dermal Hypoplasia | L-2-Hydroxyglutaric Aciduria | Hereditary Mixed Polyposis Syndrome | Anti-NMDA Receptor Encephalitis | Arts Syndrome | Muir-Torre Syndrome | Restrictive Dermopathy | Metabolic Syndrome | Alexander Disease | X-linked Charcot-Marie-Tooth Disease | Hyperlipidemia, Familial Combined | Pulmonary Vein Stenosis | Beckwith-Wiedemann Syndrome | Ependymoma | Hypertension, Renal | Tendinitis | Gaucher Disease | Maternally Inherited Diabetes And Deafness | Combined Malonic And Methylmalonic Acidemia | Pyloric Stenosis, Infantile Hypertrophic | T-cell Lymphoma, Subcutaneous Panniculitis-like | Aceruloplasminemia | Esophagitis, Eosinophilic | Acquired Partial Lipodystrophy | Basal Cell Nevus Syndrome | Pulmonary Tuberculosis | Irritable Bowel Syndrome | Dystonia-parkinsonism, X-linked | Diamond-Blackfan Anemia | DOCK8 Immunodeficiency Syndrome | Erdheim-Chester Disease | Ellis-Van Creveld Syndrome | Neurodevelopmental Disorder With Central Hypotonia And Dysmorphic Facies | Systemic Mastocytosis | Leprosy | Cardiomyopathy, Peripartum | Zollinger-Ellison Syndrome | X-linked Myotubular Myopathy | Malignant Fibrous Histiocytoma | Takayasu's Arteritis | Tangier Disease | Congenital Torticollis | Hydrocephalus | Generalized Epilepsy And Paroxysmal Dyskinesia | Hepatitis | Schnyder Crystalline Corneal Dystrophy | Glycogen Storage Disease Type 3 | Acrodermatitis | Nemaline Myopathy | Abetalipoproteinemia | Dysmorphophobia | Hydrolethalus Syndrome | Dental Caries | Arteriosclerosis | Stroke, Hemorrhagic | Dermatitis | Cheilitis | Protein C Deficiency | Wagner Disease | Multiple Sclerosis, Secondary Progressive | Acromesomelic Dysplasia | Nestor-Guillermo Progeria Syndrome | Pneumococcal Meningitis | Basan Syndrome | Charcot-Marie-Tooth Disease Type 4E | AIDS Dementia Complex | Whipple's Disease | Retinopathy Of Prematurity | Pyruvate Carboxylase Deficiency Disease | Mannosidase Deficiency Diseases | Keratoacanthoma | Pernicious Anemia | Lymphopenia | Paraplegia | CHOPS Syndrome | Cavitary Optic Disc Anomalies | Chondroma | Choreoathetosis, Hypothyroidism, And Neonatal Respiratory Distress | Gingivitis | Anthrax | Osmotic Demyelination Syndrome | Fabry's Disease | Cardiospondylocarpofacial Syndrome | Antley-Bixler Syndrome | Pupil Disorders | Periventricular Leukomalacia | Familial Digital Arthropathy-brachydactyly | Dominant Optic Atrophy