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Striatonigral degeneration, childhood-onset(SNDC)

MedGen UID:
934710
Concept ID:
C4310743
Disease or Syndrome
Synonyms: LENK-PLOSKI SYNDROME; SNDC
SNOMED CT: Childhood-onset basal ganglia degeneration syndrome (1172584005); Lenk Ploski syndrome (1172584005)
 
Gene (location): VAC14 (16q22.1-22.2)
 
Monarch Initiative: MONDO:0014889
OMIM®: 617054
Orphanet: ORPHA497906

Definition

Childhood-onset striatonigral degeneration (SNDC) is an autosomal recessive neurologic disorder characterized by sudden onset of neurodegeneration with regression of developmental milestones in the first years of life. Patients develop impaired movement with dystonia, become nonverbal and nonambulatory, and show striatal abnormalities on brain imaging (Lenk et al., 2016). [from OMIM]

Clinical features

From HPO
Ankle clonus
MedGen UID:
68672
Concept ID:
C0238651
Finding
Clonus is an involuntary tendon reflex that causes repeated flexion and extension of the foot. Ankle clonus is tested by rapidly flexing the foot upward.
Dysphagia
MedGen UID:
41440
Concept ID:
C0011168
Disease or Syndrome
Difficulty in swallowing.
Dystonic disorder
MedGen UID:
3940
Concept ID:
C0013421
Sign or Symptom
An abnormally increased muscular tone that causes fixed abnormal postures. There is a slow, intermittent twisting motion that leads to exaggerated turning and posture of the extremities and trunk.
Hyperreflexia
MedGen UID:
57738
Concept ID:
C0151889
Finding
Hyperreflexia is the presence of hyperactive stretch reflexes of the muscles.
Unsteady gait
MedGen UID:
68544
Concept ID:
C0231686
Finding
A shaky or wobbly manner of walking.
Steppage gait
MedGen UID:
98105
Concept ID:
C0427149
Finding
An abnormal gait pattern that arises from weakness of the pretibial and peroneal muscles due to a lower motor neuron lesion. Affected patients have footdrop and are unable to dorsiflex and evert the foot. The leg is lifted high on walking so that the toes clear the ground, and there may be a slapping noise when the foot strikes the ground again.
Delayed speech and language development
MedGen UID:
105318
Concept ID:
C0454644
Finding
A degree of language development that is significantly below the norm for a child of a specified age.
Developmental regression
MedGen UID:
324613
Concept ID:
C1836830
Disease or Syndrome
Loss of developmental skills, as manifested by loss of developmental milestones.
Loss of ambulation
MedGen UID:
332305
Concept ID:
C1836843
Finding
Inability to walk in a person who previous had the ability to walk.
Craniofacial dystonia
MedGen UID:
868612
Concept ID:
C4023011
Disease or Syndrome
A form of focal dystonia affecting the face and especially the jaw that is induced by the act of speaking. It is an involuntary contraction of the masticatory muscles, resulting in dysarthria or dysphagia.
Hypertonia
MedGen UID:
10132
Concept ID:
C0026826
Finding
A condition in which there is increased muscle tone so that arms or legs, for example, are stiff and difficult to move.
Hypotonia
MedGen UID:
10133
Concept ID:
C0026827
Finding
Hypotonia is an abnormally low muscle tone (the amount of tension or resistance to movement in a muscle). Even when relaxed, muscles have a continuous and passive partial contraction which provides some resistance to passive stretching. Hypotonia thus manifests as diminished resistance to passive stretching. Hypotonia is not the same as muscle weakness, although the two conditions can co-exist.
Lumbar hyperlordosis
MedGen UID:
263149
Concept ID:
C1184923
Finding
An abnormal accentuation of the inward curvature of the spine in the lumbar region.
Elevated circulating creatine kinase concentration
MedGen UID:
69128
Concept ID:
C0241005
Finding
An elevation of the level of the enzyme creatine kinase (also known as creatine phosphokinase (CK; EC 2.7.3.2) in the blood. CK levels can be elevated in a number of clinical disorders such as myocardial infarction, rhabdomyolysis, and muscular dystrophy.
Drooling
MedGen UID:
8484
Concept ID:
C0013132
Finding
Habitual flow of saliva out of the mouth.
Excessive salivation
MedGen UID:
11419
Concept ID:
C0037036
Disease or Syndrome
Excessive production of saliva.

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVStriatonigral degeneration, childhood-onset

Recent clinical studies

Etiology

Lyon GJ, Marchi E, Ekstein J, Meiner V, Hirsch Y, Scher S, Yang E, De Vivo DC, Madrid R, Li Q, Wang K, Haworth A, Chilton I, Chung WK, Velinov M
Cold Spring Harb Mol Case Stud 2019 Dec;5(6) Epub 2019 Dec 13 doi: 10.1101/mcs.a003715. PMID: 31387860Free PMC Article
Baide-Mairena H, Gaudó P, Marti-Sánchez L, Emperador S, Sánchez-Montanez A, Alonso-Luengo O, Correa M, Grau AM, Ortigoza-Escobar JD, Artuch R, Vázquez E, Del Toro M, Garrido-Pérez N, Ruiz-Pesini E, Montoya J, Bayona-Bafaluy MP, Pérez-Dueñas B
Mol Genet Metab 2019 Mar;126(3):250-258. Epub 2019 Jan 5 doi: 10.1016/j.ymgme.2019.01.001. PMID: 30642748

Prognosis

Baumann H, Tunc S, Günther A, Münchau A, Lohmann K, Brüggemann N
Parkinsonism Relat Disord 2020 Nov;80:41-46. Epub 2020 Sep 11 doi: 10.1016/j.parkreldis.2020.09.012. PMID: 32949958
Liao S, Chen T, Dai Y, Wang Y, Wu F, Zhong M
Mol Genet Genomic Med 2020 Feb;8(2):e1101. Epub 2019 Dec 26 doi: 10.1002/mgg3.1101. PMID: 31876398Free PMC Article

Clinical prediction guides

Baumann H, Tunc S, Günther A, Münchau A, Lohmann K, Brüggemann N
Parkinsonism Relat Disord 2020 Nov;80:41-46. Epub 2020 Sep 11 doi: 10.1016/j.parkreldis.2020.09.012. PMID: 32949958
Liao S, Chen T, Dai Y, Wang Y, Wu F, Zhong M
Mol Genet Genomic Med 2020 Feb;8(2):e1101. Epub 2019 Dec 26 doi: 10.1002/mgg3.1101. PMID: 31876398Free PMC Article

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