domingo, 27 de enero de 2019

Nervous System Diseases | Genetic and Rare Diseases Information Center (GARD) – an NCATS Program

Nervous System Diseases | Genetic and Rare Diseases Information Center (GARD) – an NCATS Program

National Center for Advancing and Translational Sciences

Genetic and Rare Diseases Information Center, a program of the National Center for Advancing and Translational Sciences

Nervous System Diseases



Mandibulofacial dysostosis with microcephaly 
Mannosidosis, beta A, lysosomal 
Marchiafava Bignami disease 
Marden-Walker syndrome 
Marfanoid habitus-autosomal recessive intellectual disability syndrome 
Marinesco-Sjogren syndrome 
Martsolf syndrome 
McDonough syndrome 
McLeod neuroacanthocytosis syndrome 
Meckel syndrome 
MECP2 duplication syndrome 
Medrano Roldan syndrome 
Medulloblastoma 
Megalencephalic leukoencephalopathy with subcortical cysts 
Megalencephaly-Polymicrogyria-Polydactyly-Hydrocephalus syndrome 
Megaloblastic anemia due to dihydrofolate reductase deficiency 
Megalocornea-intellectual disability syndrome 
Mehes syndrome 
Meier-Gorlin syndrome 
Meige syndrome 
Melnick-Needles syndrome 
Meningioma 
Menkes disease 
Mental retardation skeletal dysplasia abducens palsy 
Mental retardation Smith Fineman Myers type 
Mental retardation X-linked syndromic 7 
Meralgia paresthetica 
Metaphyseal dysostosis-intellectual disability-conductive deafness syndrome 
Methionine adenosyltransferase deficiency 
Methylcobalamin deficiency cbl G type 
Methylmalonic acidemia with homocystinuria type cblC 
MGAT2-CDG (CDG-IIa) 
Micro syndrome 
Microbrachycephaly ptosis cleft lip 
Microcephalic osteodysplastic primordial dwarfism type 1 
Microcephalic osteodysplastic primordial dwarfism type 2 
Microcephalic primordial dwarfism Toriello type 
Microcephalic primordial dwarfism, Montreal type 
Microcephaly 
Microcephaly autosomal dominant 
Microcephaly brain defect spasticity hypernatremia 
Microcephaly cervical spine fusion anomalies 
Microcephaly deafness syndrome 
Microcephaly glomerulonephritis Marfanoid habitus 
Microcephaly microcornea syndrome Seemanova type 
Microcephaly pontocerebellar hypoplasia dyskinesia 
Microcephaly, seizures, and developmental delay - See Early Infantile Epileptic Encephalopathy 
Microcephaly-cardiomyopathy 
Microcephaly-intellectual disability-phalangeal and neurological anomalies syndrome 
Microduplication Xp11.22-p11.23 syndrome 
Microphthalmia syndromic 10 
Microphthalmia syndromic 4 
Microphthalmia syndromic 8 
Microphthalmia with linear skin defects syndrome 
Microscopic polyangiitis 
Migraine with brainstem aura 
Mild phenylketonuria 
Miller-Dieker syndrome 
Miller-Fisher syndrome 
Minicore myopathy with external ophthalmoplegia 
Mitochondrial complex I deficiency 
Mitochondrial complex II deficiency 
Mitochondrial DNA depletion syndrome, encephalomyopathic form with methylmalonic aciduria 
Mitochondrial DNA-associated Leigh syndrome 
Mitochondrial encephalomyopathy lactic acidosis and stroke-like episodes 
Mitochondrial Membrane Protein-Associated Neurodegeneration 
Mitochondrial myopathy with diabetes 
Mitochondrial myopathy with lactic acidosis 
Mitochondrial neurogastrointestinal encephalopathy syndrome 
Mitochondrial trifunctional protein deficiency 
Mixed connective tissue disease 
Miyoshi myopathy 
Moebius syndrome 
MOGS-CDG (CDG-IIb) 
Mohr-Tranebjaerg syndrome 
Molybdenum cofactor deficiency 
Monoamine oxidase A deficiency 
Morse-Rawnsley-Sargent syndrome 
Morvan's fibrillary chorea 
Mousa Al din Al Nassar syndrome 
Moyamoya disease 
MPDU1-CDG (CDG-If) 
MPI-CDG (CDG-Ib) 
MPV17-related hepatocerebral mitochondrial DNA depletion syndrome 
Mucolipidosis type 4 
Mucopolysaccharidosis type III 
Mucopolysaccharidosis type IIIA 
Mucopolysaccharidosis type IIIB 
Mucopolysaccharidosis type IIIC 
Mucopolysaccharidosis type IIID 
Multifocal motor neuropathy 
Multiple congenital anomalies-hypotonia-seizures syndrome 
Multiple congenital anomalies-hypotonia-seizures syndrome type 2 
Multiple myeloma 
Multiple sulfatase deficiency 
Multiple system atrophy 
Multisystemic smooth muscle dysfunction syndrome 
Muscle eye brain disease 
Muscular dystrophy white matter spongiosis 
Muscular dystrophy, congenital, megaconial type 
Muscular phosphorylase kinase deficiency 
Musculocontractural Ehlers-Danlos syndrome 
Myasthenia gravis 
Myelocerebellar disorder 
Myelomeningocele 
MYH7-related scapuloperoneal myopathy 
Myhre syndrome 
Myoclonic epilepsy with ragged red fibers 
Myoclonus cerebellar ataxia deafness 
Myoclonus hereditary progressive distal muscular atrophy 
Myoclonus-dystonia 
Myoglobinuria recurrent 
Myopathy with extrapyramidal signs 
Myosin storage myopathy 
Myotonia congenita 
Myotonic dystrophy type 1 
Myotonic dystrophy type 2 
N syndrome 
Nance-Horan syndrome 
Narcolepsy 
Native American myopathy 
NBIA/DYT/PARK-PLA2G6 
Necrotizing autoimmune myopathy 
Neonatal adrenoleukodystrophy 
Neonatal meningitis 
Neonatal progeroid syndrome 
Neu Laxova syndrome 
Neuroblastoma 
Neurocutaneous melanosis 
Neurofaciodigitorenal syndrome 
Neuroferritinopathy 
Neurofibromatosis type 1 
Neurofibromatosis type 2 
Neuroleptic malignant syndrome 
Neuromyelitis optica 
Neuromyelitis optica spectrum disorder 
Neuronal ceroid lipofuscinosis 
Neuronal ceroid lipofuscinosis 10 
Neuronal ceroid lipofuscinosis 2 
Neuronal ceroid lipofuscinosis 3 
Neuronal ceroid lipofuscinosis 5 
Neuronal ceroid lipofuscinosis 6 
Neuronal ceroid lipofuscinosis 7 
Neuronal ceroid lipofuscinosis 9 
Neuronal intranuclear inclusion disease 
Neuropathy ataxia retinitis pigmentosa syndrome 
Neuropathy, distal hereditary motor, Jerash type 
Neuropathy, hereditary motor and sensory, Okinawa type 
Neuropathy, hereditary motor and sensory, Russe type 
Neutral lipid storage disease with myopathy 
Nevoid basal cell carcinoma syndrome 
New-onset refractory status epilepticus 
Nicolaides-Baraitser syndrome 
Niemann-Pick disease type A 
Niemann-Pick disease type B 
Niemann-Pick disease type C1 
Niemann-Pick disease type C2 
Non 24 hour sleep wake disorder 
Nondystrophic myotonia 
Noonan syndrome 
Normokalemic periodic paralysis 
Norrie disease 
Northern epilepsy 
Oculocerebrocutaneous syndrome 
Oculofaciocardiodental syndrome 
Oculopharyngeal muscular dystrophy 
Oculopharyngodistal myopathy 
Okamoto syndrome 
Olfactory neuroblastoma 
Oligoastrocytoma 
Oligodendroglioma 
Oliver syndrome 
Olivopontocerebellar atrophy 
Omphalocele cleft palate syndrome lethal 
OPHN1 syndrome 
Opsoclonus-myoclonus syndrome 
Optic atrophy 2 
Optic pathway glioma 
Ornithine transcarbamylase deficiency 
Orofaciodigital syndrome 1 
Orofaciodigital syndrome 10 
Orofaciodigital syndrome 2 
Orofaciodigital syndrome 3 
Orofaciodigital syndrome 4 
Orofaciodigital syndrome 5 
Orofaciodigital syndrome 6 
Orthostatic intolerance due to NET deficiency 
Osteopenia and sparse hair 
Osteoporosis-pseudoglioma syndrome 
Oto-palato-digital syndrome type 1 
Oto-palato-digital syndrome type 2 
Ouvrier Billson syndrome 
Pachygyria-intellectual disability-epilepsy syndrome 
PACS1-related syndrome 
Painful orbital and systemic neurofibromas-marfanoid habitus syndrome 
Pallidopyramidal syndrome 
Pallister W syndrome 
Pallister-Killian mosaic syndrome 
Pantothenate kinase-associated neurodegeneration 
Paralysis agitans, juvenile, of Hunt 
Paramyotonia congenita 
Parkinson disease type 3 
Parkinson disease type 9 
Paroxysmal exertion-induced dyskinesia 
Paroxysmal extreme pain disorder 
Paroxysmal hemicrania 
Paroxysmal kinesigenic choreoathetosis 
Paroxysomal nonkinesigenic dyskinesia 
Parsonage Turner syndrome 
Partington syndrome 
PCDH19-related female-limited epilepsy 
Pediatric autoimmune neuropsychiatric disorders associated with Streptococcus infections 
PEHO syndrome 
Pelizaeus-Merzbacher disease 
Periventricular heterotopia 
Periventricular leukomalacia 
Perry syndrome 
Peters plus syndrome 
Pfeiffer Mayer syndrome 
Pfeiffer Palm Teller syndrome 
PGM3-CDG 
PHACE syndrome 
Phosphoglycerate kinase deficiency 
Phosphoglycerate mutase deficiency 
Phosphoserine aminotransferase deficiency 
Photosensitive epilepsy 
Pick's disease 
Pitt-Hopkins syndrome 
Pitt-Hopkins-like syndrome 
Plasmacytoma 
Pleomorphic xanthoastrocytoma 
PMM2-CDG (CDG-Ia) 
POEMS syndrome 
Poliomyelitis 
POLR3-Related Leukodystrophy 
Polyarteritis nodosa 
Polycystic lipomembranous osteodysplasia with sclerosing leukoencephalopathy 
Polyneuropathy-intellectual disability-acromicria-premature menopause syndrome 
Pontine tegmental cap dysplasia 
Pontocerebellar hypoplasia 
Pontocerebellar hypoplasia type 1 
Pontocerebellar hypoplasia type 2 
Pontocerebellar hypoplasia type 3 
Pontocerebellar hypoplasia type 4 
Pontocerebellar hypoplasia type 5 
Pontocerebellar hypoplasia type 6 
Post Polio syndrome 
Posterior column ataxia 
Posterior column ataxia with retinitis pigmentosa 
Postnatal progressive microcephaly, seizures, and brain atrophy 
Potassium aggravated myotonia 
Potocki-Lupski syndrome 
PPM-X syndrome 
Prader-Willi habitus, osteopenia, and camptodactyly 
Primary amebic meningoencephalitis 
Primary angiitis of the central nervous system 
Primary basilar impression 
Primary carnitine deficiency 
Primary central nervous system lymphoma 
Primary Familial Brain Calcification 
Primary lateral sclerosis 
Primary melanoma of the central nervous system 
Primary orthostatic tremor 
Primary progressive aphasia 
Primrose syndrome 
Progressive bulbar palsy 
Progressive encephalomyelitis with rigidity and myoclonus 
Progressive external ophthalmoplegia, autosomal recessive 1 
Progressive hemifacial atrophy 
Progressive non-fluent aphasia 
Prolidase deficiency 
Proteus syndrome 
Proud syndrome 
Pseudoaminopterin syndrome 
Pseudocholinesterase deficiency 
Pseudoneonatal adrenoleukodystrophy 
Pseudoprogeria syndrome 
Pseudotrisomy 13 syndrome 
Pseudoxanthoma elasticum 
Pterygium colli mental retardation digital anomalies 
Pudendal Neuralgia 
Pure autonomic failure 
Pyridoxal 5'-phosphate-dependent epilepsy 
Pyridoxine-dependent epilepsy 
Pyruvate dehydrogenase phosphatase deficiency 
Qazi Markouizos syndrome 
Radiation induced brachial plexopathy 
Ramos Arroyo Clark syndrome 
Rapid-onset dystonia-parkinsonism 
Rasmussen encephalitis 
Reardon Wilson Cavanagh syndrome 
Reducing body myopathy 
Refsum disease 
Refsum disease, infantile form 
Renal dysplasia-limb defects syndrome 
Renier Gabreels Jasper syndrome 
Restless legs syndrome, susceptibility to, 1 - See Restless legs syndrome 
Restless legs syndrome, susceptibility to, 2 - See Restless legs syndrome 
Restless legs syndrome, susceptibility to, 3 - See Restless legs syndrome 
Restless legs syndrome, susceptibility to, 4 - See Restless legs syndrome 
Restless legs syndrome, susceptibility to, 5 - See Restless legs syndrome 
Restless legs syndrome, susceptibility to, 6 - See Restless legs syndrome 
Retinal arterial macroaneurysm with supravalvular pulmonic stenosis 
Retinal vasculopathy with cerebral leukodystrophy 
Rett syndrome 
Reversible cerebral vasoconstriction syndrome 
RFT1-CDG (CDG-In) 
Rhabdoid tumor 
Rhizomelic chondrodysplasia punctata type 1 
Riboflavin transporter deficiency 
Richards-Rundle syndrome 
Richieri Costa Da Silva syndrome 
Rigid spine syndrome 
Ring chromosome 10 
Ring chromosome 14 
Ring chromosome 20 
Rippling muscle disease 
RNAse T2-deficient leukoencephalopathy 
Roussy Levy syndrome 
RRM2B-related mitochondrial DNA depletion syndrome 
Ruvalcaba syndrome 
Sacral defect with anterior meningocele 
Salla disease 
Sandhoff disease 
Sandifer syndrome 
Sarcoidosis - Not a rare disease 
Say Barber Miller syndrome 
Say Meyer syndrome 
Scapuloperoneal syndrome, neurogenic, Kaeser type 
SCARF syndrome 
Schaaf-Yang syndrome 
Scheie syndrome 
Schimke immunoosseous dysplasia 
Schindler disease type 1 
Schinzel Giedion syndrome 
Schisis association 
Schizencephaly 
Schwannomatosis 
Schwartz Jampel syndrome 
Scott Bryant Graham syndrome 
Seaver Cassidy syndrome 
Seckel syndrome 
Semantic dementia 
Sensory ataxic neuropathy, dysarthria, and ophthalmoparesis 
Sepiapterin reductase deficiency 
Septo-optic dysplasia spectrum 
SeSAME syndrome 
SETBP1 disorder 
Severe congenital nemaline myopathy 
Severe intellectual disability-progressive spastic diplegia syndrome 
Severe X-linked intellectual disability, Gustavson type 
Shapiro syndrome 
Short-chain acyl-CoA dehydrogenase deficiency 
Shoulder girdle defect mental retardation familial 
Shprintzen omphalocele syndrome 
Shprintzen-Goldberg craniosynostosis syndrome 
Sialidosis type I 
Sialidosis, type II 
Sickle cell anemia 
Sideroblastic anemia and mitochondrial myopathy 
Simpson-Golabi-Behmel syndrome 
Single upper central incisor 
Sjogren-Larsson syndrome 
SLC35A1-CDG (CDG-IIf) 
SLC35A2-CDG 
SLC35C1-CDG (CDG-IIc) 
Slow-channel congenital myasthenic syndrome 
Smith-Lemli-Opitz syndrome 
Smith-Magenis syndrome 
Sneddon syndrome 
Snyder-Robinson syndrome 
Sonoda syndrome 
Spasmodic dysphonia 
Spastic ataxia Charlevoix-Saguenay type 
Spastic diplegia cerebral palsy 
Spastic diplegia infantile type 
Spastic paraplegia 1 
Spastic paraplegia 10 
Spastic paraplegia 11 
Spastic paraplegia 12 
Spastic paraplegia 13 
Spastic paraplegia 14 
Spastic paraplegia 15 
Spastic paraplegia 16 
Spastic paraplegia 17 
Spastic paraplegia 18 
Spastic paraplegia 19 
Spastic paraplegia 2 
Spastic paraplegia 23 
Spastic paraplegia 24 
Spastic paraplegia 25 
Spastic paraplegia 26 
Spastic paraplegia 29 
Spastic paraplegia 3 
Spastic paraplegia 31 
Spastic paraplegia 32 
Spastic paraplegia 39 
Spastic paraplegia 4 
Spastic paraplegia 51 
Spastic paraplegia 5A 
Spastic paraplegia 6 
Spastic paraplegia 7 
Spastic paraplegia 8 
Spastic paraplegia 9 
Spastic paraplegia facial cutaneous lesions 
Spastic paraplegia-epilepsy-intellectual disability syndrome 
Spastic paraplegia-glaucoma-intellectual disability syndrome 
Spastic tetraplegia-retinitis pigmentosa-intellectual disability syndrome 
Spastic tetraplegia-thin corpus callosum-progressive postnatal microcephaly syndrome 
Spina bifida occulta - Not a rare disease 
Spinal atrophy ophthalmoplegia pyramidal syndrome 
Spinal meningioma 
Spinal muscular atrophy 1 
Spinal muscular atrophy type 2 
Spinal muscular atrophy type 3 
Spinal shock 
Spinocerebellar ataxia 
Spinocerebellar ataxia 1 
Spinocerebellar ataxia 10 
Spinocerebellar ataxia 11 
Spinocerebellar ataxia 12 
Spinocerebellar ataxia 13 
Spinocerebellar ataxia 14 
Spinocerebellar ataxia 15 
Spinocerebellar ataxia 17 
Spinocerebellar ataxia 18 
Spinocerebellar ataxia 19 and 22 
Spinocerebellar ataxia 2 
Spinocerebellar ataxia 20 
Spinocerebellar ataxia 21 
Spinocerebellar ataxia 23 
Spinocerebellar ataxia 25 
Spinocerebellar ataxia 26 
Spinocerebellar ataxia 27 
Spinocerebellar ataxia 28 
Spinocerebellar ataxia 29 
Spinocerebellar ataxia 3 
Spinocerebellar ataxia 30 
Spinocerebellar ataxia 31 
Spinocerebellar ataxia 34 
Spinocerebellar ataxia 4 
Spinocerebellar ataxia 5 
Spinocerebellar ataxia 7 
Spinocerebellar ataxia 8 
Spinocerebellar ataxia 9 
Spinocerebellar ataxia autosomal recessive 3 
Spinocerebellar ataxia autosomal recessive 4 
Spinocerebellar ataxia autosomal recessive 5 
Spinocerebellar ataxia autosomal recessive 6 
Spinocerebellar ataxia autosomal recessive 7 
Spinocerebellar ataxia autosomal recessive 8 
Spinocerebellar ataxia autosomal recessive with axonal neuropathy 
Spinocerebellar ataxia type 6 
Spinocerebellar ataxia with dysmorphism 
Spinocerebellar ataxia X-linked type 2 
Spinocerebellar ataxia X-linked type 3 
Spinocerebellar ataxia X-linked type 4 
Spinocerebellar degeneration and corneal dystrophy 
Split hand urinary anomalies spina bifida 
Split spinal cord malformation 
Spondyloepiphyseal dysplasia congenita 
SRD5A3-CDG (CDG-Iq) 
SSR4-CDG 
Status epilepticus 
Steinfeld syndrome 
Stiff person syndrome 
Stocco dos Santos syndrome 
Striatonigral degeneration infantile 
Sturge-Weber syndrome 
Subacute sclerosing panencephalitis 
Subcortical band heterotopia 
Subependymal giant cell astrocytoma 
Subependymoma 
Succinic semialdehyde dehydrogenase deficiency 
Susac syndrome 
Symmetrical thalamic calcifications 
Tangier disease 
TANGO2-Related Metabolic Encephalopathy and Arrhythmias 
Tarlov cysts 
Tay-Sachs disease 
Tel Hashomer camptodactyly syndrome 
Telfer Sugar Jaeger syndrome 
Temple syndrome 
Temple-Baraitser syndrome 
Temporal epilepsy, familial 
Temtamy syndrome 
Tethered cord syndrome 
Thalamic degeneration symmetrical infantile 
Thalamic degeneration, symmetric infantile 
Thoracic dysplasia hydrocephalus syndrome 
Thoracic outlet syndromes - Not a rare disease 
Thyrotoxic periodic paralysis 
TMEM165-CDG (CDG-IIk) 
Toriello-Carey syndrome 
Tourette syndrome - Not a rare disease 
Tranebjaerg Svejgaard syndrome 
Transverse myelitis 
Trichinosis 
Trichorhinophalangeal syndrome type 2 
Trigeminal neuralgia 
Triosephosphate isomerase deficiency 
Triple A syndrome 
Troyer syndrome 
Tuberous sclerosis 
Tubular aggregate myopathy 
Tumefactive multiple sclerosis 
Typical congenital nemaline myopathy 
Tyrosine hydroxylase deficiency 
Tyrosinemia type 1 
Ullrich congenital muscular dystrophy 
Unverricht-Lundborg disease 
Van Benthem-Driessen-Hanveld syndrome 
Van Den Bosch syndrome 
Variant Creutzfeldt-Jakob disease 
Variegate porphyria 
Vein of Galen aneurysm 
Vici syndrome 
Viljoen Kallis Voges syndrome 
Visual snow syndrome 
VLCAD deficiency 
Vogt-Koyanagi-Harada disease 
Von Hippel-Lindau disease 
Walker-Warburg syndrome 
Weaver syndrome 
Welander distal myopathy, Swedish type 
Wernicke-Korsakoff syndrome 
West syndrome 
Whipple disease 
White matter hypoplasia-corpus callosum agenesis-intellectual disability syndrome 
Wiedemann Oldigs Oppermann syndrome 
Williams syndrome 
Wilson disease 
Wilson-Turner syndrome 
Wolf-Hirschhorn syndrome 
Wolman disease 
Woodhouse Sakati syndrome 
Worster Drought syndrome 
Wrinkly skin syndrome 
Wyburn-Mason syndrome 
X-linked Charcot-Marie-Tooth disease type 1 - See Charcot-Marie-Tooth disease 
X-linked Charcot-Marie-Tooth disease type 2 - See Charcot-Marie-Tooth disease 
X-linked Charcot-Marie-Tooth disease type 3 - See Charcot-Marie-Tooth disease 
X-linked Charcot-Marie-Tooth disease type 4 - See Charcot-Marie-Tooth disease 
X-linked Charcot-Marie-Tooth disease type 5 - See Charcot-Marie-Tooth disease 
X-linked Charcot-Marie-Tooth disease type 6 - See Charcot-Marie-Tooth disease 
X-linked complicated corpus callosum agenesis - See L1 syndrome 
X-linked complicated spastic paraplegia type 1 - See L1 syndrome 
X-linked creatine deficiency 
X-linked dystonia-parkinsonism/Lubag 
X-linked hereditary sensory and autonomic neuropathy with deafness 
X-linked intellectual disability - corpus callosum agenesis - spastic quadriparesis 
X-linked intellectual disability - short stature – obesity 
X-linked intellectual disability, Najm type 
X-linked intellectual disability, Schimke type 
X-linked intellectual disability, Siderius type 
X-linked intellectual disability, Turner type 
X-linked intellectual disability-dysmorphism-cerebral atrophy syndrome 
X-linked intellectual disability-plagiocephaly syndrome 
X-linked lissencephaly with abnormal genitalia 
X-linked myopathy with excessive autophagy 
X-linked myotubular myopathy 
X-linked non-specific intellectual disability 
X-linked periventricular heterotopia 
Xeroderma pigmentosum 
Xia-Gibbs syndrome 
XK aprosencephaly 
Zechi Ceide syndrome 
Zellweger syndrome 
ZTTK syndrome 

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