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X-linked non progressive cerebellar ataxia(SCAX5)

MedGen UID:
394718
Concept ID:
C2678048
Disease or Syndrome
Synonym: Spinocerebellar ataxia, X-linked 5
Modes of inheritance:
X-linked recessive inheritance
MedGen UID:
375779
Concept ID:
C1845977
Finding
Source: Orphanet
A mode of inheritance that is observed for recessive traits related to a gene encoded on the X chromosome. In the context of medical genetics, X-linked recessive disorders manifest in males (who have one copy of the X chromosome and are thus hemizygotes), but generally not in female heterozygotes who have one mutant and one normal allele.
 
Monarch Initiative: MONDO:0010404
OMIM®: 300703
Orphanet: ORPHA314978

Definition

X-linked non progressive cerebellar ataxia is a rare hereditary ataxia characterized by delayed early motor development, severe neonatal hypotonia, non-progressive ataxia and slow eye movements, presenting normal cognitive abilities and absence of pyramidal signs. Frequently patients also manifest intention tremor, mild dysphagia, and dysarthria. Brain MRI reveals global cerebellar atrophy with absence of other malformations or degenerations of the central and peripheral nervous systems. [from ORDO]

Clinical features

From HPO
Cerebellar ataxia
MedGen UID:
849
Concept ID:
C0007758
Disease or Syndrome
Cerebellar ataxia refers to ataxia due to dysfunction of the cerebellum. This causes a variety of elementary neurological deficits including asynergy (lack of coordination between muscles, limbs and joints), dysmetria (lack of ability to judge distances that can lead to under- or overshoot in grasping movements), and dysdiadochokinesia (inability to perform rapid movements requiring antagonizing muscle groups to be switched on and off repeatedly).
Dysarthria
MedGen UID:
8510
Concept ID:
C0013362
Mental or Behavioral Dysfunction
Dysarthric speech is a general description referring to a neurological speech disorder characterized by poor articulation. Depending on the involved neurological structures, dysarthria may be further classified as spastic, flaccid, ataxic, hyperkinetic and hypokinetic, or mixed.
Action tremor
MedGen UID:
65875
Concept ID:
C0234376
Sign or Symptom
A tremor present when the limbs are active, either when outstretched in a certain position or throughout a voluntary movement.
Motor delay
MedGen UID:
381392
Concept ID:
C1854301
Finding
A type of Developmental delay characterized by a delay in acquiring motor skills.
Intellectual disability
MedGen UID:
811461
Concept ID:
C3714756
Mental or Behavioral Dysfunction
Intellectual disability, previously referred to as mental retardation, is characterized by subnormal intellectual functioning that occurs during the developmental period. It is defined by an IQ score below 70.
Neonatal hypotonia
MedGen UID:
412209
Concept ID:
C2267233
Disease or Syndrome
Muscular hypotonia (abnormally low muscle tone) manifesting in the neonatal period.
Nystagmus
MedGen UID:
45166
Concept ID:
C0028738
Disease or Syndrome
Rhythmic, involuntary oscillations of one or both eyes related to abnormality in fixation, conjugate gaze, or vestibular mechanisms.

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVX-linked non progressive cerebellar ataxia
Follow this link to review classifications for X-linked non progressive cerebellar ataxia in Orphanet.

Recent clinical studies

Etiology

Quelle-Regaldie A, Sobrido-Cameán D, Barreiro-Iglesias A, Sobrido MJ, Sánchez L
Cells 2021 Feb 17;10(2) doi: 10.3390/cells10020421. PMID: 33671313Free PMC Article
Huang S, Zhu S, Li XJ, Li S
Neuroscientist 2019 Oct;25(5):512-520. Epub 2019 Jan 7 doi: 10.1177/1073858418822993. PMID: 30614396Free PMC Article
Fan HC, Ho LI, Chi CS, Chen SJ, Peng GS, Chan TM, Lin SZ, Harn HJ
Cell Transplant 2014;23(4-5):441-58. doi: 10.3727/096368914X678454. PMID: 24816443
Matilla-Dueñas A
Adv Exp Med Biol 2012;724:172-88. doi: 10.1007/978-1-4614-0653-2_14. PMID: 22411243
Banfi S, Zoghbi HY
Baillieres Clin Neurol 1994 Aug;3(2):281-95. PMID: 7952848

Diagnosis

Kavanaugh BC, Elacio J, Best CR, St Pierre DG, Pescosolido MF, Ouyang Q, Biedermann J, Bradley RS, Liu JS, Jones RN, Morrow EM
J Med Genet 2024 Oct 23;61(11):1031-1039. doi: 10.1136/jmg-2024-109973. PMID: 39237363Free PMC Article
Pescosolido MF, Stein DM, Schmidt M, El Achkar CM, Sabbagh M, Rogg JM, Tantravahi U, McLean RL, Liu JS, Poduri A, Morrow EM
Ann Neurol 2014 Oct;76(4):581-93. Epub 2014 Sep 19 doi: 10.1002/ana.24225. PMID: 25044251Free PMC Article
Romani M, Micalizzi A, Valente EM
Lancet Neurol 2013 Sep;12(9):894-905. Epub 2013 Jul 17 doi: 10.1016/S1474-4422(13)70136-4. PMID: 23870701Free PMC Article
Matilla-Dueñas A
Adv Exp Med Biol 2012;724:172-88. doi: 10.1007/978-1-4614-0653-2_14. PMID: 22411243
Banfi S, Zoghbi HY
Baillieres Clin Neurol 1994 Aug;3(2):281-95. PMID: 7952848

Prognosis

Ogaki K, Koga S, Aoki N, Lin W, Suzuki K, Ross OA, Dickson DW
Neuropathology 2016 Feb;36(1):64-76. Epub 2015 Jul 31 doi: 10.1111/neup.12230. PMID: 26227820Free PMC Article
Matilla-Dueñas A
Adv Exp Med Biol 2012;724:172-88. doi: 10.1007/978-1-4614-0653-2_14. PMID: 22411243
Le Ber I, Clot F, Vercueil L, Camuzat A, Viémont M, Benamar N, De Liège P, Ouvrard-Hernandez AM, Pollak P, Stevanin G, Brice A, Dürr A
Neurology 2006 Nov 28;67(10):1769-73. doi: 10.1212/01.wnl.0000244484.60489.50. PMID: 17130408
Titomanlio L, Pierri NB, Romano A, Imperati F, Borrelli M, Barletta V, Diano AA, Castaldo I, Santoro L, Del Giudice E
Am J Med Genet A 2005 Jul 15;136(2):198-200. doi: 10.1002/ajmg.a.30795. PMID: 15940696
Allikmets R, Raskind WH, Hutchinson A, Schueck ND, Dean M, Koeller DM
Hum Mol Genet 1999 May;8(5):743-9. doi: 10.1093/hmg/8.5.743. PMID: 10196363

Clinical prediction guides

Quelle-Regaldie A, Sobrido-Cameán D, Barreiro-Iglesias A, Sobrido MJ, Sánchez L
Cells 2021 Feb 17;10(2) doi: 10.3390/cells10020421. PMID: 33671313Free PMC Article
Ogaki K, Koga S, Aoki N, Lin W, Suzuki K, Ross OA, Dickson DW
Neuropathology 2016 Feb;36(1):64-76. Epub 2015 Jul 31 doi: 10.1111/neup.12230. PMID: 26227820Free PMC Article
Allikmets R, Raskind WH, Hutchinson A, Schueck ND, Dean M, Koeller DM
Hum Mol Genet 1999 May;8(5):743-9. doi: 10.1093/hmg/8.5.743. PMID: 10196363
Illarioshkin SN, Tanaka H, Markova ED, Nikolskaya NN, Ivanova-Smolenskaya IA, Tsuji S
Ann Neurol 1996 Jul;40(1):75-83. doi: 10.1002/ana.410400113. PMID: 8687195
Banfi S, Zoghbi HY
Baillieres Clin Neurol 1994 Aug;3(2):281-95. PMID: 7952848

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