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Sialidosis

MedGen UID:
120621
Concept ID:
C0268226
Disease or Syndrome
Synonyms: Cherry Red Spot Myoclonus Syndrome; Cherry Red Spot-Myoclonus Syndrome; Deficiencies, Glycoprotein Neuraminidase; Deficiency, Glycoprotein Neuraminidase; Glycoprotein Neuraminidase Deficiencies; Glycoprotein Neuraminidase Deficiency; Mucolipidoses, Type I; Mucolipidosis I; Mucolipidosis Type 1; Mucolipidosis Type I; Mucolipidosis, Type I; Myoclonus Cherry Red Spot Syndrome; Myoclonus-Cherry Red Spot Syndrome; Type I Mucolipidoses; Type I Mucolipidosis
SNOMED CT: Mucolipidosis type I (38795005); Mucolipidosis, type I (38795005); Sialidosis (38795005); Mucolipidosis I (38795005)
Modes of inheritance:
Autosomal recessive inheritance
MedGen UID:
141025
Concept ID:
C0441748
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in individuals with two pathogenic alleles, either homozygotes (two copies of the same mutant allele) or compound heterozygotes (whereby each copy of a gene has a distinct mutant allele).
 
Related gene: NEU1
 
Monarch Initiative: MONDO:0017734
OMIM®: 256550
Orphanet: ORPHA309294

Definition

A lysosomal storage disease, belonging to the group of oligosaccharidosis or with a wide clinical spectrum that is divided into two main clinical subtypes: sialidosis type I, the milder, non dysmorphic form of the disease with characteristics of gait abnormalities, progressive visual loss, bilateral macular cherry red spots and myoclonus, that presents in adolescence or adulthood (second or third decade of life); and sialidosis type II (see this term) the more severe, early onset form, with characteristics of progressive and severe mucopolysaccharidosis-like phenotype with coarse facies, visceromegaly, dysostosis multiplex, and developmental delay. Bilateral macular cherry red spots are also present. Sialidosis type II has been further divided into congenital (with hydrops fetalis), infantile and juvenile presentations. [from SNOMEDCT_US]

Professional guidelines

PubMed

Du YC, Ma LH, Li QF, Ma Y, Dong Y, Wu ZY
Orphanet J Rare Dis 2024 Sep 30;19(1):362. doi: 10.1186/s13023-024-03378-5. PMID: 39350194Free PMC Article
Al-Kouatly HB, Makhamreh MM, Rice SM, Smith K, Harman C, Quinn A, Valcarcel BN, Firman B, Liu R, Hegde M, Critchlow E, Berger SI
Genet Med 2021 Jul;23(7):1325-1333. Epub 2021 Mar 8 doi: 10.1038/s41436-021-01121-0. PMID: 33686258
Meikle PJ, Ranieri E, Simonsen H, Rozaklis T, Ramsay SL, Whitfield PD, Fuller M, Christensen E, Skovby F, Hopwood JJ
Pediatrics 2004 Oct;114(4):909-16. doi: 10.1542/peds.2004-0583. PMID: 15466084

Recent clinical studies

Etiology

Ługowska A
Cells 2024 Oct 8;13(19) doi: 10.3390/cells13191664. PMID: 39404425Free PMC Article
Minassian BA
Prog Brain Res 2014;213:113-22. doi: 10.1016/B978-0-444-63326-2.00006-5. PMID: 25194486
Girard JM, Turnbull J, Ramachandran N, Minassian BA
Handb Clin Neurol 2013;113:1731-6. doi: 10.1016/B978-0-444-59565-2.00043-5. PMID: 23622396
Zupanc ML, Legros B
Cerebellum 2004;3(3):156-71. doi: 10.1080/14734220410035356. PMID: 15543806
Meikle PJ, Hopwood JJ, Clague AE, Carey WF
JAMA 1999 Jan 20;281(3):249-54. doi: 10.1001/jama.281.3.249. PMID: 9918480

Diagnosis

Franceschetti S, Canafoglia L
Epileptic Disord 2016 Sep 1;18(S2):89-93. doi: 10.1684/epd.2016.0845. PMID: 27621198
Girard JM, Turnbull J, Ramachandran N, Minassian BA
Handb Clin Neurol 2013;113:1731-6. doi: 10.1016/B978-0-444-59565-2.00043-5. PMID: 23622396
Valayannopoulos V, Nicely H, Harmatz P, Turbeville S
Orphanet J Rare Dis 2010 Apr 12;5:5. doi: 10.1186/1750-1172-5-5. PMID: 20385007Free PMC Article
Zupanc ML, Legros B
Cerebellum 2004;3(3):156-71. doi: 10.1080/14734220410035356. PMID: 15543806
Meikle PJ, Hopwood JJ, Clague AE, Carey WF
JAMA 1999 Jan 20;281(3):249-54. doi: 10.1001/jama.281.3.249. PMID: 9918480

Therapy

van de Vlekkert D, Hu H, Weesner JA, Fremuth LE, Brown SA, Lu M, Gomero E, Campos Y, Sheppard H, d'Azzo A
Mol Ther 2024 Jul 3;32(7):2094-2112. Epub 2024 May 25 doi: 10.1016/j.ymthe.2024.05.029. PMID: 38796704Free PMC Article
Hwu WL, Chang K, Liu YH, Wang HC, Lee NC, Chien YH
Gene Ther 2024 May;31(5-6):263-272. Epub 2024 Feb 7 doi: 10.1038/s41434-024-00443-3. PMID: 38321198
Canafoglia L, Barbella G, Ferlazzo E, Striano P, Magaudda A, d'Orsi G, Martino T, Avolio C, Aguglia U, Sueri C, Giuliano L, Sofia V, Zibordi F, Ragona F, Freri E, Costa C, Nardi Cesarini E, Fanella M, Rossi Sebastiano D, Riguzzi P, Gambardella A, Di Bonaventura C, Michelucci R, Granata T, Bisulli F, Licchetta L, Tinuper P, Beccaria F, Visani E, Franceschetti S
Epilepsy Res 2019 Oct;156:106191. Epub 2019 Aug 16 doi: 10.1016/j.eplepsyres.2019.106191. PMID: 31446282
Bonesso L, Piraud M, Caruba C, Van Obberghen E, Mengual R, Hinault C
Orphanet J Rare Dis 2014 Feb 6;9:19. doi: 10.1186/1750-1172-9-19. PMID: 24502792Free PMC Article
Federico A, Battistini S, Ciacci G, de Stefano N, Gatti R, Durand P, Guazzi GC
Dev Neurosci 1991;13(4-5):320-6. doi: 10.1159/000112180. PMID: 1817038

Prognosis

Valayannopoulos V, Nicely H, Harmatz P, Turbeville S
Orphanet J Rare Dis 2010 Apr 12;5:5. doi: 10.1186/1750-1172-5-5. PMID: 20385007Free PMC Article
Seyrantepe V, Poupetova H, Froissart R, Zabot MT, Maire I, Pshezhetsky AV
Hum Mutat 2003 Nov;22(5):343-52. doi: 10.1002/humu.10268. PMID: 14517945
Meikle PJ, Hopwood JJ, Clague AE, Carey WF
JAMA 1999 Jan 20;281(3):249-54. doi: 10.1001/jama.281.3.249. PMID: 9918480
Ovali F, Samanci N, Güray A, Akdoğan Z, Akdeniz C, Dağoğlu T, Petorak I
Turk J Pediatr 1998 Jul-Sep;40(3):447-51. PMID: 9763912
Buchhalter JR
J Child Neurol 1994 Oct;9 Suppl 1:S12-9. doi: 10.1177/0883073894009001041. PMID: 7822747

Clinical prediction guides

Ługowska A
Cells 2024 Oct 8;13(19) doi: 10.3390/cells13191664. PMID: 39404425Free PMC Article
Han X, Wu S, Wang M, Li H, Huang Y, Sui R
Mol Genet Genomic Med 2020 Aug;8(8):e1316. Epub 2020 May 26 doi: 10.1002/mgg3.1316. PMID: 32453490Free PMC Article
Franceschetti S, Canafoglia L
Epileptic Disord 2016 Sep 1;18(S2):89-93. doi: 10.1684/epd.2016.0845. PMID: 27621198
Seyrantepe V, Poupetova H, Froissart R, Zabot MT, Maire I, Pshezhetsky AV
Hum Mutat 2003 Nov;22(5):343-52. doi: 10.1002/humu.10268. PMID: 14517945
Federico A, Battistini S, Ciacci G, de Stefano N, Gatti R, Durand P, Guazzi GC
Dev Neurosci 1991;13(4-5):320-6. doi: 10.1159/000112180. PMID: 1817038

Recent systematic reviews

Iyer NS, Gimovsky AC, Ferreira CR, Critchlow E, Al-Kouatly HB
Clin Genet 2021 Nov;100(5):493-503. Epub 2021 Jul 16 doi: 10.1111/cge.14005. PMID: 34057202

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