GenTIGSA Gene Database on Rare Genetic Disorders

Select a RGD Category for details 

RGDs registry under the 'Multisystemic disorders'
Disorder Name (Total=58) Aliases Sub-Category Gene Indian Reports Link
Hypoparathyroidism, deafness, renal disease syndrome
Autosomal dominant
•Barakat syndrome
•Hypoparathyroidism-sensorineural deafness-renal disease syndrome
GATA3 GATA binding protein 3
Reports
Updated as of Mar 09, 2023
PubMed
Alstrom syndrome
Autosomal recessive
•Alström Syndrome
•Alstrom's syndrome
ALMS1 ALMS1 centrosome and basal body associated protein
Reports
Updated as of Nov 30, 2023
PubMed
Mosaic variegated aneuploidy syndrome 2
Autosomal recessive
•Mosaic variegated aneuploidy syndrome
CEP57 centrosomal protein 57
Reports
Updated as of Feb 01, 2024
PubMed
Aicardi-Goutieres syndrome 6
Autosomal recessive
•Aicardi-Goutières syndrome
•Encephalopathy with basal ganglia calcification
•Encephalopathy with intracranial calcification and chronic lymphocytosis of cerebrospinal fluid
•Pseudo-Torch syndrome
•Aicardi-Toutieres syndrome
•Type 6Aicardi-Goutieres syndrome 1
ADAR adenosine deaminase RNA specific
Reports
Updated as of Jul 20, 2024
PubMed
Aicardi-Goutieres syndrome 4
Autosomal recessive
•Aicardi-Goutières syndrome
•Encephalopathy with basal ganglia calcification
•Encephalopathy with intracranial calcification and chronic lymphocytosis of cerebrospinal fluid
•Pseudo-Torch syndrome
•Aicardi-Toutieres syndrome
RNASEH2A ribonuclease H2 subunit A
Reports
Updated as of Jul 20, 2024
PubMed
Wolcott-Rallison dysplasia
Autosomal recessive
•Multiple epiphyseal dysplasia with early-onset diabetes mellitus
•Wolcott-rallison syndrome
•MED-IDDM syndrome
•Wolcott Rallison syndrome
EIF2AK3 eukaryotic translation initiation factor 2 alpha kinase 3
Reports
Updated as of Feb 01, 2025
PubMed
Hereditary angioedema type 1
Autosomal dominant, Autosomal recessive
•Deficiency of C1 esterase inhibitor
Dermatitis SERPING1 serpin family G member 1
Reports
Updated as of Feb 01, 2025
PubMed
Costello syndrome
Autosomal dominant
•Faciocutaneoskeletal syndrome
•Fcs syndrome
•congenital myopathy with excess of muscle spindles
HRAS HRas proto-oncogene, GTPase
Reports
Updated as of Feb 01, 2025
PubMed
Zellweger spectrum disorders
•Congenital iron overload
•cerebrohepatorenal syndrome
•Zellweger syndrome
•Zellweger Spectrum
•ZS
•ZWS
PEX1 peroxisomal biogenesis factor 1
PEX6 peroxisomal biogenesis factor 6
PEX19 peroxisomal biogenesis factor 19
Reports
Updated as of Feb 01, 2025
PubMed
Autosomal recessive multiple pterygium syndrome
Autosomal recessive
•Autosomal recessive multiple pterygium syndrome
•Escobar syndrome
•Pterygium Colli syndrome
•autosomal recessive non-lethal multiple pterygium syndrome
•Escobar variant multiple pterygium syndrome
•Multiple pterygium syndrome Escobar type
•Multiple pterygium syndrome nonlethal type
CHRNG cholinergic receptor nicotinic gamma subunit
CHRNA1 cholinergic receptor nicotinic alpha 1 subunit
Reports
Updated as of Feb 01, 2025
PubMed
Lethal multiple pterygium syndrome
Autosomal recessive
CHRND cholinergic receptor nicotinic delta subunit
CHRNA1 cholinergic receptor nicotinic alpha 1 subunit
CHRNG cholinergic receptor nicotinic gamma subunit
Reports
Updated as of Feb 01, 2025
PubMed
Jervell and Lange-Nielsen syndrome
Autosomal recessive
•Jervell-Lange Nielsen syndrome
•Jervell and Lange Nielsen syndrome
•long QT interval-deafness syndrome
KCNQ1 potassium voltage-gated channel subfamily Q member 1
KCNE1 potassium voltage-gated channel subfamily E regulatory subunit 1
Reports
Updated as of Feb 01, 2025
PubMed
Cerebellar ataxia-hypogonadism syndrome
Autosomal recessive
•Gordon-Holmes syndrome
•luteinizing hormone-releasing hormone deficiency with ataxia
•Cerebellar ataxia hypogonadotropic hypogonadism
•Gordon Holmes syndrome
•Lhrh deficiency and ataxia
•Luteinizing hormone releasing hormone, deficiency of with ataxia
RNF216 ring finger protein 216
Reports
Updated as of Feb 01, 2025
PubMed
Rubinstein-Taybi syndrome due to CREBBP mutations
Autosomal dominant
•Broad thumbs and great toes, characteristic facies, and mental retardation
•Rubinstein syndrome
•Rubinstein-Taybi syndrome 1, incomplete
•Rubinstein-Taybi syndrome 1
CREBBP CREB binding protein
EP300 E1A binding protein p300
Reports
Updated as of Feb 26, 2025
PubMed
Popliteal pterygium syndrome
Autosomal dominant
•Autosomal dominant popliteal pterygium syndrome
•popliteal pterygium syndrome 1
•popliteal pterygium syndrome, autosomal dominant
•popliteal web syndrome
IRF6 interferon regulatory factor 6
Reports
Updated as of Feb 26, 2025
PubMed
Muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A1
Autosomal recessive
•Cerebroocular dysgenesis
•Chemke syndrome
•COD-MD syndrome
•Cerebroocular dysplasia muscular dystrophy syndrome
•Hard +/- E syndrome
•Hydrocephalus, agyria and retinal dysplasia
•Muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A, 1
•Pagon syndrome
•WALKER-WARBURG SYNDROME OR MUSCLE-EYE-BRAIN DISEASE, POMT1-RELATED
•Warburg syndrome
POMT1 protein O-mannosyltransferase 1
Reports
Updated as of Feb 26, 2025
PubMed
Cerebrooculofacioskeletal syndrome 2
Autosomal recessive
ERCC2 ERCC excision repair 2, TFIIH core complex helicase subunit
Reports
Updated as of Feb 26, 2025
PubMed
8q24.3 microdeletion syndrome
Autosomal dominant
•Verheij syndrome
•Chromosome 8q24.3 deletion syndrome
PUF60 poly(U) binding splicing factor 60
Reports
Updated as of Sep 08, 2025
PubMed
Gorlin syndrome
Autosomal dominant
•Nevoid basal cell carcinoma syndrome
•Basal cell nevus syndrome
•Gorlin syndrome
•Gorlin-Goltz syndrome
•Multiple basal cell carcinomas
•NBCCS
•Nevoid basal cell cancer syndrome
PTCH1 patched 1
PTCH2 patched 2
SUFU SUFU negative regulator of hedgehog signaling
Reports
Updated as of Jun 12, 2025
PubMed
Zimmermann-Laband syndrome
Autosomal dominant
•Gingival fibromatosis-hepatosplenomegaly-other anomalies syndrome
•Laband syndrome
•Zimmerman Laband syndrome
•Zimmermann-Laband syndrome type 1
•ZLS
KCNH1 potassium voltage-gated channel subfamily H member 1
ATP6V1B2 ATPase H+ transporting V1 subunit B2
KCNN3 potassium calcium-activated channel subfamily N member 3
Reports
Updated as of Nov 12, 2025
PubMed
X-linked lissencephaly with abnormal genitalia
X-linked recessive
•X-linked Lissencephaly with agenesis of corpus callosum and genital anomaly syndrome
•Lissencephaly, x-linked, type 2
•X-Linked Lissencephaly with ambiguous genitalia
•x-linked lissencephaly with abnormal genitalia syndrome
•x-linked lissencephaly with ambiguous genitalia
•x-linked lissencephaly-agenesis of the corpus callosum-genital anomalies syndrome
•x-linked lissencephaly-corpus callosum agenesis-genital anomalies syndrome
•xlag (x-linked lissencephaly with abnormal genitalia) syndrome
ARX aristaless related homeobox
Reports
Updated as of Nov 12, 2025
PubMed
Dyggve-Melchior-Clausen syndrome
Autosomal recessive
•DMC syndrome
•Dyggve-Melchior-Clausen disease
•Dyggve Melchior Clausen syndrome
•Dyggve-Melchior-Clausen dysplasia
•Pseudo-Morquio disease type i
•DMC disease
DYM dymeclin
Reports
Updated as of Nov 12, 2025
PubMed
X-linked intellectual disability, van Esch type
X-linked recessive
•Van Esch-O'Driscoll syndrome
•Mental retardation, x-linked, syndromic, Van Esch-O''Driscoll type
•Van Esch-O'Driscoll syndrome, x-linked recessive
•VEODS
•x-linked intellectual disability van esch type
POLA1 DNA polymerase alpha 1, catalytic subunit
Reports
Updated as of Nov 12, 2025
PubMed
Incontinentia pigmenti syndrome
X-linked dominant
•Bloch-Sulzberger syndrome
•Bloch-Siemens syndrome
•Incontinentia Pigmenti
•Incontinentia pigmenti of Bloch-Sulzberger
•Incontinentia pigmenti syndrome
•Incontinentia pigmenti, x-linked dominant
•Incontinentia pigmenti, familial male-lethal type
•Incontinentia pigmenti, type ii
•IP - Incontinentia Pigmenti
IKBKG inhibitor of nuclear factor kappa B kinase regulatory subunit gamma
Reports
Updated as of Nov 12, 2025
PubMed
Alpha thalassemia-X-linked intellectual disability syndrome
X-linked dominant
•XLMR hypotonic face syndrome
•ATR-X syndrome
•Alpha thalassemia x-linked intellectual deficit
•Alpha thalassemia x-linked intellectual disability syndrome
•Alpha thalassemia/intellectual disability syndrome x-linked
•Alpha-thalassemia x-linked intellectual disability syndrome
•Alpha-thalassemia/impaired intellectual development syndrome, x-linked
•Alpha-thalassemia/intellectual disability syndrome nondeletion type
•Alpha-thalassemia/mental retardation syndrome, x-linked dominant
•x-linked alpha-thalassemia-mental retardation syndrome
•ATR, nondeletion type
•Alpha thalassemia mental retardation syndrome, nondeletion type, X-linked
•Alpha-Thalassemia X-Linked Intellectual Disability Syndrome
•X-linked alpha-thalassemia-mental retardation syndrome
ATRX ATRX chromatin remodeler
GBA1 glucosylceramidase beta 1
Reports
Updated as of Nov 12, 2025
PubMed
Simpson-Golabi-Behmel syndrome type 1
X-linked recessive
•Bulldog syndrome
•Simpson-Golabi-Behmel syndrome
•Dysplasia gigantism syndrome, x-linked
•Golabi-Rosen syndrome
•GPC3-related Simpson-Golabi-Behmel syndrome type 1
•Simpson dysmorphia syndrome
•Sara Angers syndrome
•x-linked dysplasia gigantism syndrome
•sgb syndrome
•dgsx
•sdys
•sgbs1
GPC3 glypican 3
Reports
Updated as of Nov 12, 2025
PubMed
Holt-Oram syndrome
Autosomal dominant
•Cardiac-limb syndrome
•Heart-hand syndrome, type 1
•TBX5-Related Holt-Oram Syndrome
•Ventriculo-radial syndrome
•Atriodigital dysplasia type 1
•Holt Oram syndrome
•HOS
TBX5 T-box transcription factor 5
Reports
Updated as of Nov 12, 2025
PubMed
AHDC1-related intellectual disability - obstructive sleep apnea - mild dysmorphism syndrome
Autosomal dominant
•Xia-Gibbs syndrome
•AHDC1-related intellectual disability-obstructive sleep apnea-mild dysmorphism syndrome
•AHDC1-related intellectual disability, obstructive sleep apnea, mild dysmorphism syndrome
•At-hook dna binding motif containing 1-related intellectual disability, obstructive sleep apnea, mild dysmorphism syndrome
•Autosomal dominant intellectual disability 25
•MRD25
AHDC1 AT-hook DNA binding motif containing 1
Reports
Updated as of Jan 05, 2026
PubMed
Arthrogryposis, renal dysfunction, and cholestasis 1
Autosomal recessive
•ARCS1
•Arthrogryposis with renal dysfunction and cholestasis syndrome
•ARC (arthrogryposis, renal dysfunction, cholestasis) syndrome
•ARC syndrome
• arthrogryposis-renal dysfunction-cholestasis
•arthrogryposis-renal dysfunction-cholestasis syndrome
•arthrogryposis, renal dysfunction and cholestasis (arc) syndrome
•arthrogryposis, renal dysfunction, and cholestasis
VPS33B VPS33B late endosome and lysosome associated
VIPAS39 VPS33B interacting protein, apical-basolateral polarity regulator, spe-39 homolog
FBN1 fibrillin 1
CPAP centrosome assembly and centriole elongation protein
Reports
Updated as of Jan 05, 2026
PubMed
Cardiospondylocarpofacial syndrome
Autosomal dominant
•Forney syndrome
•CSCF
•Forney-Robinson-pascoe syndrome
•Forney Robinson Pascoe syndrome
•Mitral regurgitation with deafness and skeletal anomalies syndrome
•Mitral regurgitation-deafness-skeletal anomalies syndrome
•Mitral regurgitation-hearing loss-skeletal anomalies syndrome
MAP3K7 mitogen-activated protein kinase kinase kinase 7
Reports
Updated as of Jan 05, 2026
PubMed
Coffin-Lowry syndrome
X-linked dominant
•Mental retardation with osteocartilaginous abnormalities
•CLS - coffin-lowry syndrome
•Coffin Lowry syndrome
•Coffin-Lowry syndrome, mild
•Coffin-Lowry syndrome, x-linked dominant
RPS6KA3 ribosomal protein S6 kinase A3
Reports
Updated as of Jan 05, 2026
PubMed
Wiedemann-Steiner syndrome
Autosomal dominant
•Growth deficiency and mental retardation with facial dysmorphism
•WDSTS
•Hypertrichosis-short stature-facial dysmorphism-developmental delay syndrome
•Hypertrichosis, short stature, facial dysmorphism, developmental delay syndrome
•Wiedemann Steiner syndrome
KMT2A lysine methyltransferase 2A
SMC1A structural maintenance of chromosomes 1A
ARID1B AT-rich interaction domain 1B
CHD7 chromodomain helicase DNA binding protein 7
Reports
Updated as of Jan 05, 2026
PubMed
Trichorhinophalangeal dysplasia type I
Autosomal dominant
•Trichorhinophalangeal syndrome type I
•Sugio-Kajii syndrome
•Trichorhinophalangeal dysplasia (syndrome) types 1/3
•Trichorhinophalangeal dysplasia type i
•Trichorhinophalangeal dysplasia types 1/3
•Trichorhinophalangeal syndrome i
•Trichorhinophalangeal syndrome type 1
•Trichorhinophalangeal syndrome, type 1
•Trichorhinophalangeal syndrome, type i
•TRPS i - trichorhinophalangeal syndrome i
•Type i trichorhinophalangeal syndrome
•Type iii trichorhinophalangeal syndrome
•Trichorhinophalangeal syndrome, Type I
•TRPS I
•TRPS1
•TRPS 1
TRPS1 transcriptional repressor GATA binding 1
Reports
Updated as of Jan 05, 2026
PubMed
Congenital anomalies of kidney and urinary tract 1
Autosomal dominant
•CAKUT1
•Congenital Anomalies of the Kidney and Urinary Tract 1
•Congenital anomalies of kidney and urinary tract 1, susceptibility to
•Renal hypodysplasia, nonsyndromic, 1
•Renal Hypodysplasia, Nonsyndromic, 1
•RHDNS
•Non-Syndromic Renal Hypodysplasia 1
DSTYK dual serine/threonine and tyrosine protein kinase
TRAP1 TNF receptor associated protein 1
CELSR3 cadherin EGF LAG seven-pass G-type receptor 3
PAX2 paired box 2
Reports
Updated as of Jan 05, 2026
PubMed
Microcephaly with or without chorioretinopathy, lymphedema, or intellectual disability
Autosomal dominant
•Chorioretinal dysplasia-microcephaly-intellectual disability syndrome
•lymphedema and retinal folds with ficrocephaly and microphthalmos
•lymphedema, microcephaly and chorioretinopathy syndrome
•microcephaly lymphedema chorioretinal dysplasia
•microcephaly with or without chorioretinopathy, lymphedema, or impaired intellectual development
•microcephaly, lymphedema, chorioretinal dysplasia syndrome
•mlcrd
•Lymphedema, Microcephaly, Chorioretinopathy Syndrome
•MCLMR
•Microcephaly and chorioretinopathy with or without mental retardation, autosomal dominant
•Microcephaly with or without chorioretinopathy, lymphedema, or mental retardation
KIF11 kinesin family member 11
Reports
Updated as of Jan 05, 2026
PubMed
Neonatal ichthyosis-sclerosing cholangitis syndrome
Autosomal recessive
•Ichthyosis-hypotrichosis-sclerosing cholangitis syndrome
•Ichthyosis, hypotrichosis, sclerosing cholangitis syndrome
•IHSC
•Neonatal sclerosing cholangitis, ichthyosis, hypotrichosis syndrome
•Nisch (neonatal ichthyosis, sclerosing cholangitis, hypotrichosis) syndrome
•Ichthyosis-sclerosing cholangitis syndrome
•ILVASC
•NISCH syndrome
CLDN1 claudin 1
Reports
Updated as of Jan 05, 2026
PubMed
Coffin-Siris syndrome 1
Autosomal dominant
•ARID1B-related bafopathy
•ARID1B-related Coffin-Siris syndrome
•Hypertrichosis, H
•CSS1
•Mental retardation, autosomal dominant 12
ARID1B AT-rich interaction domain 1B
SOX4 SRY-box transcription factor 4
SMARCD1 SWI/SNF related BAF chromatin remodeling complex subunit D1
BICRA BRD4 interacting chromatin remodeling complex associated protein
SMARCC2 SWI/SNF related BAF chromatin remodeling complex subunit C2
SOX11 SRY-box transcription factor 11
Reports
Updated as of Feb 10, 2026
PubMed
Frasier syndrome
Autosomal dominant
•Frasier syndrome, autosomal dominant, somatic mutation
•Syndrome, Frasier
WT1 WT1 transcription factor
Reports
Updated as of Feb 10, 2026
PubMed
KBG syndrome
Autosomal dominant
•Short stature-characteristic facies-mental retardation-macrodontia-skeletal anomalies syndrome
•Short stature-facial and skeletal anomalies-intellectual disability-macrodontia syndrome
•Short stature, facial and skeletal anomalies, intellectual disability, macrodontia syndrome
•KBGS
ANKRD11 ankyrin repeat domain 11
KAT6B lysine acetyltransferase 6B
SETD5 SET domain containing 5
TBX1 T-box transcription factor 1
COL2A1 collagen type II alpha 1 chain
Reports
Updated as of Feb 10, 2026
PubMed
Temtamy syndrome
Autosomal recessive
•Craniofacial dysmorphism with coloboma of eye and corpus callosum agenesis syndrome
•Craniofacial dysmorphism-coloboma-corpus callosum agenesis syndrome
•Temtamy Shalash syndrome
•Temtamy-Shalash syndrome
•Mental retardation with or without craniofacial dysmorphism, ocular coloboma, or abnormal corpus callosum
•TEMTYS
C12orf57 chromosome 12 open reading frame 57
Reports
Updated as of Feb 10, 2026
PubMed
CHARGE syndrome
Autosomal dominant
•CHD7-related CHARGE syndrome
•CHARGE syndrome due to CHD7 deficiency
•Hall-Hittner syndrome
•Hall Hittner syndrome
•Hittner Hirsch Kreh syndrome
•CHARGE association
•CHARGE
•CHARGE ASSOCIATION--COLOBOMA, HEART ANOMALY, CHOANAL ATRESIA, RETARDATION, GENITAL AND EAR ANOMALIES
•Coloboma, heart anomaly, choanal atresia, retardation, genital and ear anomalies
•Coloboma, heart defects, choanal atresia, retardation of growth and development, genitourinary problems, ear abnormalities syndrome
•Coloboma, heart defects, choanal atresia, retardation of growth and development, genital abnormalities, and ear anomalies association
•Coloboma, heart malformation, choanal atresia, retardation of growth and development, genital abnormalities, and ear malformations (charge) association
•Coloboma, heart malformation, choanal atresia, retardation of growth and development, genital abnormalities, and ear malformations association
•Coloboma, heart defects, choanal atresia, retardation of growth and development, genitourinary problems, ear abnormalities syndrome
•coloboma-heart defects-atresia choanae-retardation of growth and development-genitourinary problems-ear abnormalities syndrome
•Colobomatous microphthalmia, heart disease, hearing loss, and mental retardation
•Coloboma, congenital heart disease, choanal atresia, growth retardation, genital hypoplasia, ear and hearing anomaly syndrome
•CHARGE (coloboma, heart anomaly, choanal atresia, retardation, genital and ear anomalies) syndrome
CHD7 chromodomain helicase DNA binding protein 7
SEMA3E semaphorin 3E
TNFRSF1A TNF receptor superfamily member 1A
Reports
Updated as of Feb 10, 2026
PubMed
Cornelia de Lange syndrome 1
Autosomal dominant
•NIPBL-Related Cornelia de Lange Syndrome
•Brachmann de Lange syndrome
•Typus degenerativus amstelodamensis
•Degenerative amsterodamensis typus
•Cornelia de Lange syndrome
•Amsterdam dwarf
•Brachmann-de Lange syndrome
•Bruck-de Lange syndrome
•de Lange syndrome
•CDLS1
NIPBL NIPBL cohesin loading factor
TAF6 TATA-box binding protein associated factor 6
KMT2A lysine methyltransferase 2A
HDAC8 histone deacetylase 8
SMC3 structural maintenance of chromosomes 3
RAD21 RAD21 cohesin complex component
Reports
Updated as of Feb 10, 2026
PubMed
Hermansky-Pudlak syndrome 4
Autosomal recessive
•HPS4
HPS4 HPS4 biogenesis of lysosomal organelles complex 3 subunit 2
Reports
Updated as of Feb 10, 2026
PubMed
Arthrogryposis, renal dysfunction, and cholestasis 2
Autosomal recessive
•VIPAS39-Related Arthrogryposis, Renal Dysfunction, and Cholestasis Syndrome
•ARC syndrome type 2
•ARCS2
•Arthrogryposis with renal dysfunction and cholestasis 2 syndrome
VIPAS39 VPS33B interacting protein, apical-basolateral polarity regulator, spe-39 homolog
Reports
Updated as of Feb 10, 2026
PubMed
CATARACTS, SPASTIC PARAPARESIS, AND SPEECH DELAY
Autosomal dominant
•Fatty acyl-CoA reductase 1 superactivity
•Spastic paraparesis, cataracts, speech delay syndrome
•CSPSD
•Spastic Paraparesis-Cataracts-Speech Delay Syndrome
FAR1 fatty acyl-CoA reductase 1
Reports
Updated as of Feb 10, 2026
PubMed
Mitochondrial DNA depletion syndrome 1
Autosomal recessive
•MNGIE, TYMP-related
•Mitochondrial neurogastrointestinal encephalopathy syndrome, tymp-related
•Mitochondrial DNA depletion syndrome 1 (MNGIE type)
•Mitochondrial DNA Depletion Syndrome, MNGIE Form
•Mitochondrial neurogastrointestinal encephalomyopathy syndrome
•Mitochondrial Neurogastrointestinal Encephalopathy Disease
•MTDPS1
•POLIP syndrome
•Polyneuropathy, ophthalmoplegia, leukoencephalopathy, and intestinal pseudoobstruction syndrome
MT-TK mitochondrially encoded tRNA lysine
TYMP thymidine phosphorylase
SLC25A4 solute carrier family 25 member 4
MGME1 mitochondrial genome maintenance exonuclease 1
FBXL4 F-box and leucine rich repeat protein 4
TFAM transcription factor A, mitochondrial
OPA1 OPA1 mitochondrial dynamin like GTPase
MRM2 mitochondrial rRNA methyltransferase 2
SLC25A21 solute carrier family 25 member 21
SLC25A10 solute carrier family 25 member 10
Reports
Updated as of Feb 10, 2026
PubMed
Hermansky-Pudlak syndrome 10
Autosomal recessive
•HPS10
AP3D1 adaptor related protein complex 3 subunit delta 1
Reports
Updated as of Mar 30, 2026
PubMed
Radial aplasia-thrombocytopenia syndrome
Autosomal recessive
•Thrombocytopenia-absent radius syndrome
•1q21.1 susceptibility locus for thrombocytopenia-absent radius (tar) syndrome
•TAR - thrombocytopenia with absent radius syndrome
• thrombocytopenia with absent radii (tar) syndrome
•Thrombocytopenia with absent radius syndrome
•Thrombocytopenia-absent radii syndrome
•TAR syndrome
•Thrombocytopenia Absent Radius Syndrome
•TAR - Thrombocytopenia with absent radius syndrome
•Radial aplasia-thrombocytopenia syndrome
•TAR
RBM8A RNA binding motif protein 8A
Reports
Updated as of Mar 30, 2026
PubMed
Renal tubular acidosis with progressive nerve deafness
Autosomal recessive
•Distal renal tubular acidosis with progressive sensorineural deafness
•Renal tubular acidosis, autosomal recessive, with progressive nerve deafness
•RTA with progressive nerve deafness
•Renal tubular acidosis, distal, 2, with progressive sensorineural hearing loss
•Renal tubular acidosis with progressive nerve deafness
•Renal tubular acidosis with deafness
•Autosomal recessive distal renal tubular acidosis with deafness
•AR dRTA (autosomal recessive distal renal tubular acidosis) with deafness
•Autosomal recessive distal RTA
•AR dRTA
•Autosomal recessive distal renal tubular acidosisAutosomal recessive distal renal tubular acidosis (disease)
•Autosomal recessive distal RTA
ATP6V1B1 ATPase H+ transporting V1 subunit B1
Reports
Updated as of Mar 30, 2026
PubMed
Hermansky-Pudlak syndrome 1
Autosomal recessive
•Storage pool disease of platelets
•Delta storage pool disease
•HPS1
•Alpha dense granule deficiency
•Combined alpha-delta platelet storage pool deficiency
•Dense body defect
•Dense body deficiency
•Nucleotide storage pool disorder
•Platelet dense granule deficiency
•Platelet storage organelle defect
•Platelet storage pool defect
•Platelet storage pool deficiency
•Storage pool deficiency
HPS1 HPS1 biogenesis of lysosomal organelles complex 3 subunit 1
AP3D1 adaptor related protein complex 3 subunit delta 1
BLOC1S5 biogenesis of lysosomal organelles complex 1 subunit 5
ETV6 ETS variant transcription factor 6
Reports
Updated as of Mar 30, 2026
PubMed
Vici syndrome
Autosomal recessive
•Absent corpus callosum-cataract-immunodeficiency syndrome
•Corpus callosum agenesis-cataract-immunodeficiency syndrome
•Dionisi-Vici-Sabetta-Gambarara syndrome
•Dionisi Vici Sabetta Gambarara syndrome
•VICIS
EPG5 ectopic P-granules 5 autophagy tethering factor
Reports
Updated as of Mar 30, 2026
PubMed
Cardiofaciocutaneous syndrome 3
Autosomal dominant
•MAP2K1-Related Cardiofaciocutaneous Syndrome
•CFC3
MAP2K1 mitogen-activated protein kinase kinase 1
Reports
Updated as of Mar 30, 2026
PubMed
ZTTK syndrome
Autosomal dominant
•SON -Related Syndrome
•Zttk multiple congenital anomalies-mental retardation syndrome
•Brain malformations, musculoskeletal abnormalities, facial dysmorphism, intellectual disability syndrome
•Zhu Tokita Takenouchi Kim syndrome
•Zhu-Tokita-Takenouchi-Kim Syndrome
•TOKIMS
•ZTTKS
•Tokita-Kim syndrome
SON SON DNA and RNA binding protein
Reports
Updated as of Apr 28, 2026
PubMed
Young syndrome
Autosomal recessive
•Barry-Perkins-Young syndrome
•Azoospermia-sinopulmonary infections syndrome
•Azoospermia, obstructive, and chronic sinopulmonary infections
•Ciliary dyskinesia, primary, 55
•Sinusitis-infertility syndrome
•CILD55
•Young's syndrome
CFAP221 cilia and flagella associated protein 221
Reports
Updated as of Apr 28, 2026
PubMed
Woodhouse-Sakati syndrome
Autosomal recessive
•Diabetes-hypogonadism-deafness-intellectual disability syndrome
•Diabetes-hypogonadism-hearing loss-intellectual disability syndrome
•Diabetes, hypogonadism, deafness, intellectual disability syndrome
•Hypogonadism, alopecia, diabetes mellitus, mental retardation, and extrapyramidal syndrome
•Hypogonadism, diabetes mellitus, alopecia, mental retardation and electrocardiographic abnormalities
•Extrapyramidal disorder, progressive, with primary hypogonadism, mental retardation, and alopecia
•Woodhouse Sakati syndrome
DCAF17 DDB1 and CUL4 associated factor 17
Reports
Updated as of Apr 28, 2026
PubMed
Scalp-ear-nipple syndrome
Autosomal dominant
•Finlay marks syndrome
•Hereditary syndrome of lumpy scalp, odd ears and rudimentary nipples
•Finlay-marks syndrome
•Scalp, ear, nipple syndrome
•SEN syndrome
•SENS
KCTD1 potassium channel tetramerization domain containing 1
Reports
Updated as of Apr 28, 2026
PubMed
ACCES syndrome
Autosomal dominant
•Aplasia cutis congenita with ectrodactyly skeletal syndrome
UBA2 ubiquitin like modifier activating enzyme 2
Reports
Updated as of Apr 28, 2026
PubMed
Hypoparathyroidism-retardation-dysmorphism syndrome
Autosomal recessive
•Sanjad Sakati syndrome
•Hypoparathyroidism with short stature, intellectual disability and seizures
•Hypoparathyroidism-intellectual disability-dysmorphism syndrome
•Hypoparathyroidism-retardation-dysmorphism syndrome
•Hypoparathyroidism-short stature-intellectual disability-seizures syndrome
•Hypoparathyroidism, intellectual disability, dysmorphism syndrome
•Hypoparathyroidism, short stature, intellectual disability, seizures syndrome
•Richardson Kirk syndrome
•Richardson-Kirk syndrome
•Sanjad-Sakati syndrome
•SSS - Sanjad Sakati syndrome
•SSS
•HRDS
TBCE tubulin folding cofactor E
Reports
Updated as of Apr 28, 2026
PubMed