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Neuronal ceroid lipofuscinosis

MedGen UID:
10326
Concept ID:
C0027877
Disease or Syndrome
Synonym: Ceroid storage disease
SNOMED CT: Neuronal ceroid lipofuscinosis (42012007); Cerebromacular degeneration (42012007); Cerebromacular dystrophy (42012007); Pigmentary retinal lipoid neuronal heredodegeneration (42012007)
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).
Autosomal dominant inheritance
MedGen UID:
141047
Concept ID:
C0443147
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 heterozygotes. In the context of medical genetics, an autosomal dominant disorder is caused when a single copy of the mutant allele is present. Males and females are affected equally, and can both transmit the disorder with a risk of 50% for each child of inheriting the mutant allele.
 
Related genes: MFSD8, DNAJC5, CLN6, PPT1, CLN8, CTSD, CLN5, CLN3, TPP1
 
Monarch Initiative: MONDO:0016295
OMIM® Phenotypic series: PS256730
Orphanet: ORPHA216

Definition

CLN2 disease is one of a group of disorders known as neuronal ceroid lipofuscinoses (NCLs), which may also be collectively referred to as Batten disease. All these disorders affect the nervous system and typically cause worsening problems with vision, movement, and thinking ability. The different NCLs are distinguished by their genetic cause. Each disease type is given the designation "CLN," meaning ceroid lipofuscinosis, neuronal, and then a number to indicate its subtype.

Some children with CLN2 disease do not develop symptoms until later in childhood, typically after age 4. These individuals tend to have milder features overall compared to those diagnosed earlier, but with more severe ataxia. They have a shortened life expectancy, although they tend to survive into adulthood.

CLN2 disease is an inherited disorder that primarily affects the nervous system. The signs and symptoms of this condition typically begin between ages 2 and 4. The initial features usually include recurrent seizures (epilepsy) and difficulty coordinating movements (ataxia). Affected children also develop muscle twitches (myoclonus) and vision loss. CLN2 disease affects motor skills, such as sitting and walking, and speech development. This condition also causes the loss of previously acquired skills (developmental regression), intellectual disability that gradually gets worse, and behavioral problems. Individuals with this condition often require the use of a wheelchair by late childhood and typically do not survive past their teens. [from MedlinePlus Genetics]

Term Hierarchy

Follow this link to review classifications for Neuronal ceroid lipofuscinosis in Orphanet.

Professional guidelines

PubMed

Simon MJ, Logan T, DeVos SL, Di Paolo G
Trends Cell Biol 2023 Apr;33(4):324-339. Epub 2022 Oct 13 doi: 10.1016/j.tcb.2022.09.006. PMID: 36244875
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Recent clinical studies

Etiology

Rhinn H, Tatton N, McCaughey S, Kurnellas M, Rosenthal A
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Bras JM
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Diagnosis

Mohapatra A, Sabir M, Shaikh N
Ophthalmol Retina 2023 Jan;7(1):58. Epub 2022 Nov 18 doi: 10.1016/j.oret.2022.10.016. PMID: 36411198
Gundersen CB
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Ashrafi MR, Amanat M, Garshasbi M, Kameli R, Nilipour Y, Heidari M, Rezaei Z, Tavasoli AR
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Mastrangelo M
Acta Neurol Belg 2019 Dec;119(4):511-521. Epub 2019 Jun 3 doi: 10.1007/s13760-019-01160-0. PMID: 31161467
Goebel HH
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Therapy

Schulz A, Specchio N, de Los Reyes E, Gissen P, Nickel M, Trivisano M, Aylward SC, Chakrapani A, Schwering C, Wibbeler E, Westermann LM, Ballon DJ, Dyke JP, Cherukuri A, Bondade S, Slasor P, Cohen Pfeffer J
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Prognosis

Galosi S, Edani BH, Martinelli S, Hansikova H, Eklund EA, Caputi C, Masuelli L, Corsten-Janssen N, Srour M, Oegema R, Bosch DGM, Ellis CA, Amlie-Wolf L, Accogli A, Atallah I, Averdunk L, Barañano KW, Bei R, Bagnasco I, Brusco A, Demarest S, Alaix AS, Di Bonaventura C, Distelmaier F, Elmslie F, Gan-Or Z, Good JM, Gripp K, Kamsteeg EJ, Macnamara E, Marcelis C, Mercier N, Peeden J, Pizzi S, Pannone L, Shinawi M, Toro C, Verbeek NE, Venkateswaran S, Wheeler PG, Zdrazilova L, Zhang R, Zorzi G, Guerrini R, Sessa WC, Lefeber DJ, Tartaglia M, Hamdan FF, Grabińska KA, Leuzzi V
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Deneubourg C, Ramm M, Smith LJ, Baron O, Singh K, Byrne SC, Duchen MR, Gautel M, Eskelinen EL, Fanto M, Jungbluth H
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Cooper JD, Mole SE
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Clinical prediction guides

Saleh MM, Hamhom AM, Al-Otaibi A, AlGhamdi M, Housawi Y, Aljadhai YI, Alameer S, Almannai M, Jad LA, Alwadei AH, Tabassum S, Alsaman A, AlAsmari A, Al Mutairi F, Althiyab H, Bashiri FA, AlHumaidi S, Alfadhel M, Mink JW, AlHashim A, Faqeih EA; Saudi NCL Study Consortium
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Schulz A, Specchio N, de Los Reyes E, Gissen P, Nickel M, Trivisano M, Aylward SC, Chakrapani A, Schwering C, Wibbeler E, Westermann LM, Ballon DJ, Dyke JP, Cherukuri A, Bondade S, Slasor P, Cohen Pfeffer J
Lancet Neurol 2024 Jan;23(1):60-70. doi: 10.1016/S1474-4422(23)00384-8. PMID: 38101904
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Recent systematic reviews

Quintas S, Sanles-Falagan R, Berbís MÁ
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