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Lethal arthrogryposis with anterior horn cell disease

MedGen UID:
394833
Concept ID:
C2678471
Disease or Syndrome
Synonym: Lethal Arthrogryposis With Anterior Horn Cell Disease
SNOMED CT: Vuopala disease (715565004); Lethal arthrogryposis co-occurrent with anterior horn cell disease (715565004); Lethal arthrogryposis with anterior horn cell disease (715565004)

Definition

A rare arthrogryposis syndrome characterized by the association of arthrogryposis multiplex congenita and a severe form of motor neuron disease with loss of anterior horn cells in the spinal cord. Patients present with fetal akinesia deformation sequence with multiple contractures and facial anomalies, such as low-set ears, hypoplastic jaw, and short neck, as well as hypotonia and respiratory insufficiency. Some patients may survive into childhood and show developmental delay, markedly decreased muscle bulk, dystonic and involuntary movements, ataxia, and poor speech. [from ORPHANET]

Recent clinical studies

Prognosis

Paakkola T, Vuopala K, Kokkonen H, Ignatius J, Valkama M, Moilanen JS, Fahiminiya S, Majewski J, Hinttala R, Uusimaa J
Clin Genet 2018 Jan;93(1):173-177. Epub 2017 Nov 24 doi: 10.1111/cge.13086. PMID: 28657126
Said E, Chong JX, Hempel M, Denecke J, Soler P, Strom T, Nickerson DA, Kubisch C; University of Washington Center for Mendelian Genomics, Bamshad MJ, Lessel D
Am J Med Genet A 2017 Nov;173(11):3098-3103. Epub 2017 Sep 8 doi: 10.1002/ajmg.a.38406. PMID: 28884921Free PMC Article
Folkmann AW, Dawson TR, Wente SR
Adv Biol Regul 2014 Jan;54:74-91. Epub 2013 Nov 13 doi: 10.1016/j.jbior.2013.10.002. PMID: 24275432Free PMC Article

Clinical prediction guides

Said E, Chong JX, Hempel M, Denecke J, Soler P, Strom T, Nickerson DA, Kubisch C; University of Washington Center for Mendelian Genomics, Bamshad MJ, Lessel D
Am J Med Genet A 2017 Nov;173(11):3098-3103. Epub 2017 Sep 8 doi: 10.1002/ajmg.a.38406. PMID: 28884921Free PMC Article
Folkmann AW, Dawson TR, Wente SR
Adv Biol Regul 2014 Jan;54:74-91. Epub 2013 Nov 13 doi: 10.1016/j.jbior.2013.10.002. PMID: 24275432Free PMC Article

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