U.S. flag

An official website of the United States government

Format

Send to:

Choose Destination

Pontocerebellar atrophy

MedGen UID:
381261
Concept ID:
C1853766
Disease or Syndrome; Finding
Synonym: Cerebellopontine atrophy
 
HPO: HP:0006879

Definition

Atrophy affecting the pons and the cerebellum. [from HPO]

Term Hierarchy

Conditions with this feature

Congenital disorder of glycosylation type 1E
MedGen UID:
324784
Concept ID:
C1837396
Disease or Syndrome
Congenital disorders of glycosylation (CDGs) are metabolic deficiencies in glycoprotein biosynthesis that usually cause severe mental and psychomotor retardation. Different forms of CDGs can be recognized by altered isoelectric focusing (IEF) patterns of serum transferrin. For a general discussion of CDGs, see CDG Ia (212065) and CDG Ib (602579).
Spinocerebellar ataxia, autosomal recessive, with axonal neuropathy 2
MedGen UID:
340052
Concept ID:
C1853761
Disease or Syndrome
Ataxia with oculomotor apraxia type 2 (AOA2) is characterized by onset of ataxia between age three and 30 years after initial normal development, axonal sensorimotor neuropathy, oculomotor apraxia, cerebellar atrophy, and elevated serum concentration of alpha-fetoprotein (AFP).
Spinocerebellar ataxia type 40
MedGen UID:
1385103
Concept ID:
C4518336
Disease or Syndrome
A very rare disease with characteristics of adult-onset unsteady gait and dysarthria, followed by wide-based gait, gait ataxia, ocular dysmetria, intention tremor, scanning speech, hyperreflexia and dysdiadochokinesis.
Intellectual disability, autosomal dominant 56
MedGen UID:
1638835
Concept ID:
C4693389
Mental or Behavioral Dysfunction
Intellectual developmental disorder with or without epilepsy or cerebellar ataxia
MedGen UID:
1648354
Concept ID:
C4748041
Disease or Syndrome
Cerebellar hypoplasia-intellectual disability-congenital microcephaly-dystonia-anemia-growth retardation syndrome
MedGen UID:
1780242
Concept ID:
C5543287
Disease or Syndrome
CIMDAG syndrome (CIMDAG) is a multisystemic disorder characterized by severely impaired psychomotor development and hematologic abnormalities apparent from early infancy. Affected individuals show poor overall growth with microcephaly, impaired intellectual development, poor or absent speech, poor eye contact, and motor problems, such as inability to walk, hypotonia, and spasticity. Brain imaging typically shows cerebral and cerebellar atrophy, thin corpus callosum, and delayed myelination. The associated hematologic abnormalities are variable, but are mostly consistent with congenital dyserythropoietic anemia (CDA) (summary by Rodger et al., 2020 and Seu et al., 2020).
Neurodevelopmental disorder with motor and language delay, ocular defects, and brain abnormalities
MedGen UID:
1841232
Concept ID:
C5830596
Disease or Syndrome
Neurodevelopmental disorder with motor and language delay, ocular defects, and brain abnormalities (NEDMLOB) is an autosomal recessive neurologic disorder characterized by the onset of features in infancy or early childhood. Affected individuals show hypotonia, severe motor delay with ataxic gait or sometimes an inability to achieve walking, and impaired intellectual development with speech and language delay. Ocular defects can include optic atrophy, nystagmus, strabismus, and retinal dystrophy. Additional features may include seizures (in some), dysmorphic facial features, poor overall growth, and variable brain imaging abnormalities (Tepe et al., 2023).

Professional guidelines

PubMed

Tohyama J, Nakashima M, Nabatame S, Gaik-Siew C, Miyata R, Rener-Primec Z, Kato M, Matsumoto N, Saitsu H
J Hum Genet 2015 Apr;60(4):167-73. Epub 2015 Jan 29 doi: 10.1038/jhg.2015.5. PMID: 25631096

Recent clinical studies

Etiology

Májovská J, Nestrašil I, Ahmed A, Bondy MT, Klempíř J, Jahnová H, Schneider SA, Horáková D, Krásenský J, Ješina P, Vaneckova M, Nascene DR, Whitley CB, Jarnes JR, Magner M, Dušek P
J Inherit Metab Dis 2024 Mar;47(2):327-339. Epub 2023 Dec 19 doi: 10.1002/jimd.12700. PMID: 38112342Free PMC Article
Ngoh A, Bras J, Guerreiro R, Meyer E, McTague A, Dawson E, Mankad K, Gunny R, Clayton P, Mills PB, Thornton R, Lai M, Forsyth R, Kurian MA
Epilepsia 2016 May;57(5):e97-e102. Epub 2016 Apr 8 doi: 10.1111/epi.13358. PMID: 27061686Free PMC Article
Mascalchi M, Diciotti S, Giannelli M, Ginestroni A, Soricelli A, Nicolai E, Aiello M, Tessa C, Galli L, Dotti MT, Piacentini S, Salvatore E, Toschi N
PLoS One 2014;9(2):e89410. Epub 2014 Feb 25 doi: 10.1371/journal.pone.0089410. PMID: 24586758Free PMC Article
Rabiah PK, Bateman JB, Demer JL, Perlman S
Am J Ophthalmol 1997 Jan;123(1):108-17. doi: 10.1016/s0002-9394(14)71000-1. PMID: 9186105
Bawle EV, Kupsky WJ, D'Amato CJ, Becker CJ, Hicks S
Pediatr Neurol 1995 Jul;13(1):14-8. doi: 10.1016/0887-8994(95)00081-p. PMID: 7575842

Diagnosis

Ramadesikan S, Hickey S, De Los Reyes E, Patel AD, Franklin SJ, Brennan P, Crist E, Lee K, White P, McBride KL, Koboldt DC, Wilson RK
Cold Spring Harb Mol Case Stud 2022 Feb;8(2) Epub 2022 Mar 24 doi: 10.1101/mcs.a006165. PMID: 35091508Free PMC Article
Májovská J, Hennig A, Nestrasil I, Schneider SA, Jahnová H, Vaněčková M, Magner M, Dušek P
Neurol Sci 2022 May;43(5):3273-3281. Epub 2021 Nov 20 doi: 10.1007/s10072-021-05757-3. PMID: 34800199
Tohyama J, Nakashima M, Nabatame S, Gaik-Siew C, Miyata R, Rener-Primec Z, Kato M, Matsumoto N, Saitsu H
J Hum Genet 2015 Apr;60(4):167-73. Epub 2015 Jan 29 doi: 10.1038/jhg.2015.5. PMID: 25631096
Sanefuji M, Kira R, Matsumoto K, Gondo K, Torisu H, Kawakami H, Iwaki T, Hara T
J Child Neurol 2010 Nov;25(11):1429-34. Epub 2010 Jun 17 doi: 10.1177/0883073810372991. PMID: 20558670
Rabiah PK, Bateman JB, Demer JL, Perlman S
Am J Ophthalmol 1997 Jan;123(1):108-17. doi: 10.1016/s0002-9394(14)71000-1. PMID: 9186105

Therapy

Rodríguez-Labrada R, Canales-Ochoa N, Galicia-Polo ML, Cruz-Rivas E, Romanzetti S, Peña-Acosta A, Estupiñán-Rodríguez A, Vázquez-Mojena Y, Dogan I, Auburger G, Reetz K, Velázquez-Pérez L
Cerebellum 2024 Oct;23(5):1839-1847. Epub 2024 Mar 4 doi: 10.1007/s12311-024-01674-1. PMID: 38438827
Simon NG, Parratt JD, Barnett MH, Buckland ME, Gupta R, Hayes MW, Masters LT, Reddel SW
J Neurol Neurosurg Psychiatry 2012 Jan;83(1):15-22. Epub 2011 Nov 5 doi: 10.1136/jnnp-2011-301054. PMID: 22056964
Ubhi K, Rockenstein E, Mante M, Inglis C, Adame A, Patrick C, Whitney K, Masliah E
J Neurosci 2010 May 5;30(18):6236-46. doi: 10.1523/JNEUROSCI.0567-10.2010. PMID: 20445049Free PMC Article

Prognosis

Ramadesikan S, Hickey S, De Los Reyes E, Patel AD, Franklin SJ, Brennan P, Crist E, Lee K, White P, McBride KL, Koboldt DC, Wilson RK
Cold Spring Harb Mol Case Stud 2022 Feb;8(2) Epub 2022 Mar 24 doi: 10.1101/mcs.a006165. PMID: 35091508Free PMC Article
Májovská J, Hennig A, Nestrasil I, Schneider SA, Jahnová H, Vaněčková M, Magner M, Dušek P
Neurol Sci 2022 May;43(5):3273-3281. Epub 2021 Nov 20 doi: 10.1007/s10072-021-05757-3. PMID: 34800199
Syrbe S, Harms FL, Parrini E, Montomoli M, Mütze U, Helbig KL, Polster T, Albrecht B, Bernbeck U, van Binsbergen E, Biskup S, Burglen L, Denecke J, Heron B, Heyne HO, Hoffmann GF, Hornemann F, Matsushige T, Matsuura R, Kato M, Korenke GC, Kuechler A, Lämmer C, Merkenschlager A, Mignot C, Ruf S, Nakashima M, Saitsu H, Stamberger H, Pisano T, Tohyama J, Weckhuysen S, Werckx W, Wickert J, Mari F, Verbeek NE, Møller RS, Koeleman B, Matsumoto N, Dobyns WB, Battaglia D, Lemke JR, Kutsche K, Guerrini R
Brain 2017 Sep 1;140(9):2322-2336. doi: 10.1093/brain/awx195. PMID: 29050398Free PMC Article
Sanefuji M, Kira R, Matsumoto K, Gondo K, Torisu H, Kawakami H, Iwaki T, Hara T
J Child Neurol 2010 Nov;25(11):1429-34. Epub 2010 Jun 17 doi: 10.1177/0883073810372991. PMID: 20558670
Bawle EV, Kupsky WJ, D'Amato CJ, Becker CJ, Hicks S
Pediatr Neurol 1995 Jul;13(1):14-8. doi: 10.1016/0887-8994(95)00081-p. PMID: 7575842

Clinical prediction guides

Májovská J, Nestrašil I, Ahmed A, Bondy MT, Klempíř J, Jahnová H, Schneider SA, Horáková D, Krásenský J, Ješina P, Vaneckova M, Nascene DR, Whitley CB, Jarnes JR, Magner M, Dušek P
J Inherit Metab Dis 2024 Mar;47(2):327-339. Epub 2023 Dec 19 doi: 10.1002/jimd.12700. PMID: 38112342Free PMC Article
Ramadesikan S, Hickey S, De Los Reyes E, Patel AD, Franklin SJ, Brennan P, Crist E, Lee K, White P, McBride KL, Koboldt DC, Wilson RK
Cold Spring Harb Mol Case Stud 2022 Feb;8(2) Epub 2022 Mar 24 doi: 10.1101/mcs.a006165. PMID: 35091508Free PMC Article
Syrbe S, Harms FL, Parrini E, Montomoli M, Mütze U, Helbig KL, Polster T, Albrecht B, Bernbeck U, van Binsbergen E, Biskup S, Burglen L, Denecke J, Heron B, Heyne HO, Hoffmann GF, Hornemann F, Matsushige T, Matsuura R, Kato M, Korenke GC, Kuechler A, Lämmer C, Merkenschlager A, Mignot C, Ruf S, Nakashima M, Saitsu H, Stamberger H, Pisano T, Tohyama J, Weckhuysen S, Werckx W, Wickert J, Mari F, Verbeek NE, Møller RS, Koeleman B, Matsumoto N, Dobyns WB, Battaglia D, Lemke JR, Kutsche K, Guerrini R
Brain 2017 Sep 1;140(9):2322-2336. doi: 10.1093/brain/awx195. PMID: 29050398Free PMC Article
Tohyama J, Nakashima M, Nabatame S, Gaik-Siew C, Miyata R, Rener-Primec Z, Kato M, Matsumoto N, Saitsu H
J Hum Genet 2015 Apr;60(4):167-73. Epub 2015 Jan 29 doi: 10.1038/jhg.2015.5. PMID: 25631096
Mascalchi M, Diciotti S, Giannelli M, Ginestroni A, Soricelli A, Nicolai E, Aiello M, Tessa C, Galli L, Dotti MT, Piacentini S, Salvatore E, Toschi N
PLoS One 2014;9(2):e89410. Epub 2014 Feb 25 doi: 10.1371/journal.pone.0089410. PMID: 24586758Free PMC Article

Supplemental Content

Table of contents

    Clinical resources

    Practice guidelines

    • PubMed
      See practice and clinical guidelines in PubMed. The search results may include broader topics and may not capture all published guidelines. See the FAQ for details.

    Consumer resources

    Recent activity

    Your browsing activity is empty.

    Activity recording is turned off.

    Turn recording back on

    See more...