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PRRT2-associated paroxysmal movement disorder

MedGen UID:
1053958
Concept ID:
CN377744
Disease or Syndrome
Monarch Initiative: MONDO:0100556

Definition

A group of rare movement and seizure disorders caused by changes (disease-causing variants or mutations) in the PRRT2 gene. They include a spectrum of specific disorders including paroxysmal kinesigenic dyskinesia (PKD), benign familial infantile epilepsy (BFIE), paroxysmal kinesigenic dyskinesia with infantile convulsions (PKD/IC) and hemiplegic migraine (HM). In addition, PRRT2 pathogenic variants have been identified in other childhood-onset movement disorders and different types of seizure conditions, such as paroxysmal torticollis, episodic ataxia and familial paroxysmal non-kinesigenic dyskinesia. It’s important to note that these disorders can also have different genetic causes. [from MONDO]

Professional guidelines

PubMed

De Gusmao CM, Silveira-Moriyama L
Expert Rev Neurother 2019 Sep;19(9):807-822. Epub 2019 Aug 8 doi: 10.1080/14737175.2019.1648211. PMID: 31353980

Recent clinical studies

Diagnosis

Dekker MCJ, Chengo R, Kumburu HH, Kamsteeg EJ, Hamel BC
Tremor Other Hyperkinet Mov (N Y) 2019;10 Epub 2020 Jan 10 doi: 10.7916/tohm.v0.742. PMID: 32002278Free PMC Article
De Gusmao CM, Silveira-Moriyama L
Expert Rev Neurother 2019 Sep;19(9):807-822. Epub 2019 Aug 8 doi: 10.1080/14737175.2019.1648211. PMID: 31353980
Ebrahimi-Fakhari D, Saffari A, Westenberger A, Klein C
Brain 2015 Dec;138(Pt 12):3476-95. Epub 2015 Nov 23 doi: 10.1093/brain/awv317. PMID: 26598493
Ebrahimi-Fakhari D, Kang KS, Kotzaeridou U, Kohlhase J, Klein C, Assmann BE
Neurology 2014 Oct 28;83(18):1680-3. doi: 10.1212/WNL.0000000000000936. PMID: 25349275

Therapy

Dekker MCJ, Chengo R, Kumburu HH, Kamsteeg EJ, Hamel BC
Tremor Other Hyperkinet Mov (N Y) 2019;10 Epub 2020 Jan 10 doi: 10.7916/tohm.v0.742. PMID: 32002278Free PMC Article
De Gusmao CM, Silveira-Moriyama L
Expert Rev Neurother 2019 Sep;19(9):807-822. Epub 2019 Aug 8 doi: 10.1080/14737175.2019.1648211. PMID: 31353980

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