Congenital Myasthenic Syndrome

About the Disease
Congenital Myasthenic Syndrome, also known as congenital myasthenia, is related to myasthenic syndrome, congenital, 4c, associated with acetylcholine receptor deficiency and myasthenic syndrome, congenital, 5, and has symptoms including facial paresis An important gene associated with Congenital Myasthenic Syndrome is CHRNE (Cholinergic Receptor Nicotinic Epsilon Subunit), and among its related pathways/superpathways are Transmission across Chemical Synapses and Postsynaptic nicotinic acetylcholine receptors. The drugs Racephedrine and Adrenergic Agents have been mentioned in the context of this disorder. Affiliated tissues include eye, skin and skeletal muscle, and related phenotypes are ptosis and dysphagia

Common Targets
MUSK | COLQ | MYO9A | ALG2 | COL13A1 | CHRND | AGRN | PLEC | SLC18A3 | CHRNG | CHRNE | GFPT1 | ALPK1 | DOK7 | DNM2 | POGZ | SCN4A | SYT2 | CHAT | CHRNB1 | LAMA1 | SLC5A7 | SLC25A1 | DPAGT1 | ALG14 | Potassium Channels (nonspecified subtype) | DOCK7 | AK9 | GMPPB | LRP4 | CHRNA1 | RAPSN | VAMP1 | LRP6 | LAMA5

Note: If you'd like to get a target analysis report for Congenital Myasthenic Syndrome, 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 Congenital Myasthenic Syndrome at a cost 90% lower than traditional approaches, please feel free to contact us at BD@silexon.tech.

Other Diseases

Left Ventricular Noncompaction | Beckwith-Wiedemann Syndrome | Budd-Chiari Syndrome | Pulmonary Alveolar Proteinosis | Usher Syndrome Type II | Spinocerebellar Ataxia Type 42 | Leishmaniasis, Visceral | Skin Papilloma | Non-proliferative Diabetic Retinopathy | Acute Tubular Necrosis | Rhizomelic Chondrodysplasia Punctata | Chromosome 8q21.11 Deletion Syndrome | Pontocerebellar Hypoplasia | Epidermolysis Bullosa Simplex, Dowling-Meara Type | Subacute Sclerosing Panencephalitis | Multiple Sulfatase Deficiency | Arterial Tortuosity Syndrome | Guillain-Barre Syndrome | Hyperekplexia | Uremia | Nutrition Disorders | Sclerocornea | Anosmia, Congenital | Angioedema | Burn-McKeown Syndrome | Cone Dystrophy | Urea Cycle Disorder | Familial Episodic Pain Syndrome | Hypotension, Orthostatic | Enterocolitis, Necrotizing | Renal Dysplasia | Intestinal Obstruction | Heterotaxy | Carey-Fineman-Ziter Syndrome | Wieacker-Wolff Syndrome | Malignant Fibrous Histiocytoma | Postpartum Depression | Parapsoriasis | Common Cold | 3-hydroxy-3-methylglutaryl-CoA Synthase Deficiency | Epidermolysis Bullosa Acquisita | Neuronal Ceroid Lipofuscinosis | Epithelioid Hemangioma | Basal Cell Nevus Syndrome | Hypophosphatasia | Dystonia-parkinsonism, X-linked | Clouston Hidrotic Ectodermal Dysplasia | Scapuloperoneal Myopathy, X-linked Dominant | Amenorrhea | Spinocerebellar Ataxia Type 17 | Congenital Bile Acid Synthesis Defect | Acromesomelic Dysplasia | Niemann-Pick Disease, Type B | Congenital Dyserythropoietic Anemia Type 4 | Sleep Apnea, Obstructive | Encephalopathy, Ethylmalonic | Peripheral T-cell Lymphoma | Conduct Disorder | Genitopatellar Syndrome | Giant Axonal Neuropathy | Non-bullous Congenital Ichthyosiform Erythroderma | Panic Disorder | Hyperuricemic Nephropathy, Familial Juvenile | Neuroma | Antiphospholipid Syndrome | Neuroblastoma | Glutaric Aciduria Type 3 | Epilepsy Of Infancy With Migrating Focal Seizures | Mitochondrial Cytopathy | Cardiofaciocutaneous Syndrome | Cerebral Amyloid Angiopathy | Retinopathy Of Prematurity | Schindler Disease | Pyruvate Kinase Deficiency | Esophagitis, Eosinophilic | Frontometaphyseal Dysplasia | Neovascular Glaucoma | Inflammatory Joint Disease | Microphthalmia | Inflammatory Bowel Disease | Erysipelas | Arthrogryposis | Senior-Loken Syndrome | Chromosome 5q Deletion Syndrome | Schuurs-Hoeijmakers Syndrome | Fascioliasis | Epicondylitis | Pitt-Hopkins Syndrome | Schwannoma | LEOPARD Syndrome | Cystitis | Schnitzler Syndrome | Cannabis Abuse | Abetalipoproteinemia | Spinocerebellar Ataxia Type 23 | Paraganglioma | Spondylometaphyseal Dysplasia | Li-Fraumeni Syndrome | Rubeosis Iridis | Autosomal Recessive Spastic Paraplegia Type 54 | Carcinoid Syndrome | GATA2 Deficiency | Juvenile Xanthogranuloma | Onchocerciasis | Endocarditis | Mitochondrial DNA Depletion Syndrome, Encephalomyopathic Form | Hydronephrosis | Common Variable Immunodeficiency | Diffuse Palmoplantar Keratoderma | Mood Disorder | Diffuse Intrinsic Pontine Glioma | Dysequilibrium Syndrome | Insulinoma | Cardiomyopathy, Restrictive | Cryoglobulinemia | Primary Torsion Dystonia | Lymphangioleiomyomatosis | Maternally Inherited Diabetes And Deafness | Filariasis | Hemoglobinopathies | Ventricular Septal Defect | Neuromuscular Disorders | Erdheim-Chester Disease | Donnai-Barrow Syndrome | Bartsocas-Papas Syndrome | Waardenburg Syndrome Type 2E | Tremor | Raynaud Phenomenon | 3C Syndrome | Idiopathic Pulmonary Fibrosis | Nicotine Addiction | Multicentric Carpotarsal Osteolysis Syndrome | Paronychia | Periodic Limb Movement Disorder | Acute Anterior Uveitis | Creatine Deficiency Syndrome Due To AGAT Deficiency | Myoclonic Atonic Epilepsy | Charcot-Marie-Tooth Disease, Type 2 | Familial Hyperaldosteronism | Hartsfield Syndrome | X-linked Sideroblastic Anemia | Glioblastoma | Hypokalemia | Ameloblastic Carcinoma | Diabetes Mellitus, Transient Neonatal | Hypomyelination With Atrophy Of Basal Ganglia And Cerebellum | Fibromyalgia | Tuberculous Meningitis | Angiodysplasia | Adenylosuccinate Lyase Deficiency | Renal Hypouricemia | Heart Failure | Trichorhinophalangeal Syndrome | Hemangioendothelioma | Relapsing Polychondritis | Venous Insufficiency | Specific Granule Deficiency | Leber Congenital Amaurosis | Pouchitis | Salla Disease | Vitamin A Deficiency | Microvillus Inclusion Disease | Wolman Disease | Zollinger-Ellison Syndrome | Sponastrime Dysplasia | Primary Progressive Aphasia | Speech Disorders | Rothmund-Thomson Syndrome | Postaxial Polydactyly | Tyrosinemia Type 1 | Neurofibromatosis | Cole-Carpenter Syndrome | Parkinson Disease 6, Autosomal Recessive Early-onset | Ghosal Syndrome | Sorsby Fundus Dystrophy | Methemoglobinemia | Leiomyoma | Pemphigoid | Adenomatoid Tumor | Glycogen Storage Disease Type 1b | Patent Foramen Ovale | Warsaw Breakage Syndrome | Congenital Muscular Dystrophy | Multiple Myeloma | Hypotrichosis Simplex | Paraplegia | Cerebellofaciodental Syndrome | Charcot-Marie-Tooth Disease Type 4D | Eclampsia | Hemochromatosis | Hypogammaglobulinemia | Gyrate Atrophy Of The Choroid And Retina | Analgesia | Combined Pituitary Hormone Deficiency | Miyoshi Myopathy | Swine Influenza | Dermatitis Herpetiformis | Brenner Tumor | Familial Isolated Hyperparathyroidism | Dupuytren Disease | Hyperlipidemia Type V | Withdrawal Syndrome | Leprosy | Waldenstrom Macroglobulinemia | Vasculitis | Hemorrhagic Disorders | Urolithiasis | Hypophosphatemic Rickets With Hypercalciuria, Hereditary | Impetigo | Cyclic Vomiting Syndrome | Oligospermia | Angioimmunoblastic T-cell Lymphoma | Hemochromatosis Type 2 | Ganglioneuroma | Benign Recurrent Intrahepatic Cholestasis 1 | Chiari Malformation Type I | Long QT Syndrome Type 2 | Diffuse Idiopathic Pulmonary Neuroendocrine Cell Hyperplasia | Tylosis With Esophageal Cancer | Mesial Temporal Lobe Epilepsy With Hippocampal Sclerosis | Colitis | Otopalatodigital Syndrome Type 2 | Pyruvate Carboxylase Deficiency Disease | Anorectal Fistula | Camurati-Engelmann Disease | Autism | Early Infantile Epileptic Encephalopathy 4 | Sulfite Oxidase Deficiency | Myosin Storage Myopathy | Leukocyte Adhesion Deficiency Type 1 | Familial Partial Lipodystrophy | Hypotrichosis, Congenital, With Juvenile Macular Dystrophy | Hypertelorism | Hypertension, Essential | Jawad Syndrome | Focal Facial Dermal Dysplasia | Progressive External Ophthalmoplegia | Congenital Hereditary Endothelial Dystrophy Type I | Fucosidosis | Spinocerebellar Ataxia | Lichen Planus | Blastoma, Pleuropulmonary | Carcinoma In Situ | Optic Neuritis | Non-Langerhans Cell Histiocytosis | Aneurysm, Thoracic Aortic | Alzheimer Disease, Late Onset | Encephalopathy, Hepatic | Oligoasthenoteratozoospermia | T-cell Lymphoma, Subcutaneous Panniculitis-like | Herpes Genitalis | Lyme Disease | Neurodevelopmental Disorder With Central Hypotonia And Dysmorphic Facies | Aromatic L-amino Acid Decarboxylase Deficiency | Lymphedema-distichiasis Syndrome | Cholera | Atherosclerosis | Long QT Syndrome Type 3 | Situs Inversus | Pupil Disorders | Traboulsi Syndrome | Epilepsy, Generalized | Osteoporosis | Behavioral Variant Of Frontotemporal Dementia | Bernard-Soulier Syndrome | Kawasaki Disease | Agranulocytosis | Narcolepsy | Amyotrophic Lateral Sclerosis | Bleeding Disorder Due To CalDAG-GEFI Deficiency | Epidermolytic Ichthyosis, Annular | Liddle Syndrome | Cancer, Prostate | Primary Aldosteronism | Spastic Paraplegia Type 7 | Cancer, Bladder | Spondylocostal Dysostosis | Tinea | Charcot-Marie-Tooth Disease Type 2D | Rett Syndrome | Schizencephaly | Multiple Sclerosis | Tumoral Calcinosis | Hyperparathyroidism | Corneal Edema | Reflex Epilepsy | Osteoarthritis | Aplastic Anemia | Ganglioglioma | Enhanced S-cone Syndrome | Paroxysmal Kinesigenic Dyskinesia | Hyperandrogenemia | Spinocerebellar Ataxia Type 1 | Gestational Trophoblastic Disease | Alpha-thalassemia Myelodysplasia Syndrome | Metachromatic Leukodystrophy | Generalized Epilepsy With Febrile Seizures Plus | Atrial Septal Defect | Charcot-Marie-Tooth Disease Type 4E | Blepharo-cheilo-odontic Syndrome | Central Retinal Artery Occlusion | Blood Protein Disorders | Progressive Familial Intrahepatic Cholestasis | Perivascular Epithelioid Cell Tumor | Pierre Robin Syndrome | Triphalangeal Thumb-polysyndactyly Syndrome | Cancer, Brain | Amebiasis | Kindler Syndrome | Primary Lateral Sclerosis | Oguchi Disease-2 | Hemolytic Anemia | Bare Lymphocyte Syndrome | Cavitary Optic Disc Anomalies | Cancer, Lung | Lamellar Ichthyosis | Acute Lung Injury | Spondyloarthritis | Diverticulitis | Liver Failure | Non-Hodgkin Lymphoma | Epidermolysis Bullosa Simplex | Gerstmann-Straussler-Scheinker Syndrome | Optic Atrophy 2 | Rheumatoid Arthritis | Multiple Mitochondrial Dysfunctions Syndrome Type 1 | Seborrheic Dermatitis | Panniculitis | Nestor-Guillermo Progeria Syndrome | Batten Disease | Myelitis | Photosensitivity | Pancytopenia | Progressive Encephalopathy-optic Atrophy Syndrome | Diamond-Blackfan Anemia | Actinomycetoma | Tonsillitis | Interstitial Lung Diseases | Schizotypal Personality Disorder | Thyroid Dyshormonogenesis | Nephroblastoma | Intestinal Hypomagnesemia 1 | 3-methylcrotonyl-CoA Carboxylase Deficiency | Chondroma | Osteogenesis Imperfecta Type V | Pontocerebellar Hypoplasia Type 2 | Pleurisy | Gallstones | Pantothenate Kinase-associated Neurodegeneration | Acromicric Dysplasia | Temtamy Preaxial Brachydactyly Syndrome | Endometrial Hyperplasia | Cancer, Kidney | Duane Retraction Syndrome | Cushing Syndrome | Okihiro Syndrome | Posterior Polar Cataract | LMNA-related Congenital Muscular Dystrophy | Small Lymphocytic Lymphoma | Conjunctivitis | Adenosine Deaminase 2 Deficiency | Crohn's Disease | Congenital Nephrotic Syndrome | 17-beta-hydroxysteroid Dehydrogenase X Deficiency | Oculopharyngeal Muscular Dystrophy | Ophthalmia, Sympathetic | Iron Deficiency Anemia | Ulcerative Colitis | Ataxia-hypogonadism-choroidal Dystrophy Syndrome | Anemia | Cardiac Sarcoidosis | Progressive Familial Intrahepatic Cholestasis Type 1 | Lymphangioma | Melanoma, Malignant | Peeling Skin Syndrome, Acral Type | Sick Sinus Syndrome 1 | Amish Infantile Epilepsy Syndrome | Aneurysm, Abdominal Aortic | Absence Epilepsy | Aceruloplasminemia | Thrombasthenia | Leukoencephalopathy, Progressive Multifocal | Congenital Dyserythropoietic Anemia Type 1 | Adrenomyeloneuropathy | Myotonic Disorders | Proctitis | Infertility | Aphasia | Chronic Neutrophilic Leukemia | Retinoblastoma | Desbuquois Syndrome | Holoprosencephaly | Hereditary Inclusion Body Myopathy | Pheochromocytoma | Vogt-Koyanagi-Harada Syndrome | Gigantism | Inflammatory Myofibroblastic Tumor | Lipoma | Corneal Dystrophy And Perceptive Deafness | Antley-Bixler Syndrome