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Gm2-gangliosidosis, juvenile

MedGen UID:
541260
Concept ID:
C0268276
Disease or Syndrome
Synonym: GM2-GANGLIOSIDOSIS, JUVENILE
SNOMED CT: GM>2< gangliosidosis, type 3 (9537004); Juvenile GM2 gangliosidosis (9537004)
 
OMIM®: 606869

Professional guidelines

PubMed

Mansouri V, Tavasoli AR, Khodarahmi M, Dakkali MS, Daneshfar S, Ashrafi MR, Heidari M, Hosseinpour S, Sharifianjazi F, Bemanalizadeh M
Eur J Neurol 2023 Sep;30(9):2919-2945. Epub 2023 Jun 8 doi: 10.1111/ene.15871. PMID: 37209042
Zhang J, Chen H, Kornreich R, Yu C
Methods Mol Biol 2019;1885:233-250. doi: 10.1007/978-1-4939-8889-1_16. PMID: 30506202
Lemos M, Pinto R, Ribeiro G, Ribeiro H, Lopes L, Sá Miranda MC
Prenat Diagn 1995 Jun;15(6):585-8. doi: 10.1002/pd.1970150614. PMID: 7659693

Recent clinical studies

Etiology

Kern J, Böhringer J, Timmann D, Trollmann R, Stendel C, Kamm C, Röbl M, Santhanakumaran V, Groeschel S, Beck-Wödl S, Göricke S, Krägeloh-Mann I, Synofzik M
Neurology 2024 Jan 9;102(1):e207898. Epub 2023 Dec 12 doi: 10.1212/WNL.0000000000207898. PMID: 38165373Free PMC Article
Mansouri V, Tavasoli AR, Khodarahmi M, Dakkali MS, Daneshfar S, Ashrafi MR, Heidari M, Hosseinpour S, Sharifianjazi F, Bemanalizadeh M
Eur J Neurol 2023 Sep;30(9):2919-2945. Epub 2023 Jun 8 doi: 10.1111/ene.15871. PMID: 37209042
Blondel A, Kraoua I, Marcelino C, Khrouf W, Schlemmer D, Ganne B, Caillaud C, Fernández-Eulate G, Turki IBY, Dauriat B, Bonnefont-Rousselot D, Nadjar Y, Lamari F
Mol Genet Metab 2023 Feb;138(2):106983. Epub 2022 Dec 26 doi: 10.1016/j.ymgme.2022.106983. PMID: 36709536
Shibuya M, Uneoka S, Onuma A, Kodama K, Endo W, Okubo Y, Inui T, Togashi N, Nakashima I, Hino-Fukuyo N, Ida H, Miyatake S, Matsumoto N, Haginoya K
Brain Dev 2021 Nov;43(10):1029-1032. Epub 2021 Jul 1 doi: 10.1016/j.braindev.2021.06.007. PMID: 34217565
Zhang J, Chen H, Kornreich R, Yu C
Methods Mol Biol 2019;1885:233-250. doi: 10.1007/978-1-4939-8889-1_16. PMID: 30506202

Diagnosis

Kern J, Böhringer J, Timmann D, Trollmann R, Stendel C, Kamm C, Röbl M, Santhanakumaran V, Groeschel S, Beck-Wödl S, Göricke S, Krägeloh-Mann I, Synofzik M
Neurology 2024 Jan 9;102(1):e207898. Epub 2023 Dec 12 doi: 10.1212/WNL.0000000000207898. PMID: 38165373Free PMC Article
Mansouri V, Tavasoli AR, Khodarahmi M, Dakkali MS, Daneshfar S, Ashrafi MR, Heidari M, Hosseinpour S, Sharifianjazi F, Bemanalizadeh M
Eur J Neurol 2023 Sep;30(9):2919-2945. Epub 2023 Jun 8 doi: 10.1111/ene.15871. PMID: 37209042
Sakurai M, Azuma J, Hamada Y, Yamamoto T, Sakai N
Pediatr Int 2019 Jun;61(6):611-613. doi: 10.1111/ped.13848. PMID: 31242539
Menkes JH, O'Brien JS, Okada S, Grippo J, Andrews JM, Cancilla PA
Arch Neurol 1971 Jul;25(1):14-22. doi: 10.1001/archneur.1971.00490010024003. PMID: 5146406
Suzuki K, Rapin I, Suzuki Y, Ishii N
Neurology 1970 Feb;20(2):190-204. doi: 10.1212/wnl.20.2.190. PMID: 5460705

Therapy

Kern J, Böhringer J, Timmann D, Trollmann R, Stendel C, Kamm C, Röbl M, Santhanakumaran V, Groeschel S, Beck-Wödl S, Göricke S, Krägeloh-Mann I, Synofzik M
Neurology 2024 Jan 9;102(1):e207898. Epub 2023 Dec 12 doi: 10.1212/WNL.0000000000207898. PMID: 38165373Free PMC Article
Mansouri V, Tavasoli AR, Khodarahmi M, Dakkali MS, Daneshfar S, Ashrafi MR, Heidari M, Hosseinpour S, Sharifianjazi F, Bemanalizadeh M
Eur J Neurol 2023 Sep;30(9):2919-2945. Epub 2023 Jun 8 doi: 10.1111/ene.15871. PMID: 37209042
Maegawa GH, Banwell BL, Blaser S, Sorge G, Toplak M, Ackerley C, Hawkins C, Hayes J, Clarke JT
Mol Genet Metab 2009 Sep-Oct;98(1-2):215-24. Epub 2009 Jun 12 doi: 10.1016/j.ymgme.2009.06.005. PMID: 19595619
Maegawa GH, van Giersbergen PL, Yang S, Banwell B, Morgan CP, Dingemanse J, Tifft CJ, Clarke JT
Mol Genet Metab 2009 Aug;97(4):284-91. Epub 2009 May 3 doi: 10.1016/j.ymgme.2009.04.013. PMID: 19447653
Sakuragawa N, Yoshikawa H, Sasaki M
Brain Dev 1992 Jan;14(1):7-11. doi: 10.1016/s0387-7604(12)80272-5. PMID: 1590532

Prognosis

Kern J, Böhringer J, Timmann D, Trollmann R, Stendel C, Kamm C, Röbl M, Santhanakumaran V, Groeschel S, Beck-Wödl S, Göricke S, Krägeloh-Mann I, Synofzik M
Neurology 2024 Jan 9;102(1):e207898. Epub 2023 Dec 12 doi: 10.1212/WNL.0000000000207898. PMID: 38165373Free PMC Article
Blondel A, Kraoua I, Marcelino C, Khrouf W, Schlemmer D, Ganne B, Caillaud C, Fernández-Eulate G, Turki IBY, Dauriat B, Bonnefont-Rousselot D, Nadjar Y, Lamari F
Mol Genet Metab 2023 Feb;138(2):106983. Epub 2022 Dec 26 doi: 10.1016/j.ymgme.2022.106983. PMID: 36709536
Shibuya M, Uneoka S, Onuma A, Kodama K, Endo W, Okubo Y, Inui T, Togashi N, Nakashima I, Hino-Fukuyo N, Ida H, Miyatake S, Matsumoto N, Haginoya K
Brain Dev 2021 Nov;43(10):1029-1032. Epub 2021 Jul 1 doi: 10.1016/j.braindev.2021.06.007. PMID: 34217565
Maegawa GH, Stockley T, Tropak M, Banwell B, Blaser S, Kok F, Giugliani R, Mahuran D, Clarke JT
Pediatrics 2006 Nov;118(5):e1550-62. Epub 2006 Oct 2 doi: 10.1542/peds.2006-0588. PMID: 17015493Free PMC Article
Trop I, Kaplan F, Brown C, Mahuran D, Hechtman P
Hum Mutat 1992;1(1):35-9. doi: 10.1002/humu.1380010106. PMID: 1301189

Clinical prediction guides

Kern J, Böhringer J, Timmann D, Trollmann R, Stendel C, Kamm C, Röbl M, Santhanakumaran V, Groeschel S, Beck-Wödl S, Göricke S, Krägeloh-Mann I, Synofzik M
Neurology 2024 Jan 9;102(1):e207898. Epub 2023 Dec 12 doi: 10.1212/WNL.0000000000207898. PMID: 38165373Free PMC Article
Mansouri V, Tavasoli AR, Khodarahmi M, Dakkali MS, Daneshfar S, Ashrafi MR, Heidari M, Hosseinpour S, Sharifianjazi F, Bemanalizadeh M
Eur J Neurol 2023 Sep;30(9):2919-2945. Epub 2023 Jun 8 doi: 10.1111/ene.15871. PMID: 37209042
Han ST, Hirt A, Nicoli ER, Kono M, Toro C, Proia RL, Tifft CJ
J Inherit Metab Dis 2023 Jul;46(4):687-694. Epub 2023 Feb 5 doi: 10.1002/jimd.12596. PMID: 36700853Free PMC Article
Maegawa GH, Stockley T, Tropak M, Banwell B, Blaser S, Kok F, Giugliani R, Mahuran D, Clarke JT
Pediatrics 2006 Nov;118(5):e1550-62. Epub 2006 Oct 2 doi: 10.1542/peds.2006-0588. PMID: 17015493Free PMC Article
Trop I, Kaplan F, Brown C, Mahuran D, Hechtman P
Hum Mutat 1992;1(1):35-9. doi: 10.1002/humu.1380010106. PMID: 1301189

Recent systematic reviews

Mansouri V, Tavasoli AR, Khodarahmi M, Dakkali MS, Daneshfar S, Ashrafi MR, Heidari M, Hosseinpour S, Sharifianjazi F, Bemanalizadeh M
Eur J Neurol 2023 Sep;30(9):2919-2945. Epub 2023 Jun 8 doi: 10.1111/ene.15871. PMID: 37209042
Autti T, Joensuu R, Aberg L
Neuroradiology 2007 Jul;49(7):571-8. Epub 2007 Mar 3 doi: 10.1007/s00234-007-0220-6. PMID: 17334752

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