000892509 001__ 892509 000892509 005__ 20210930133534.0 000892509 0247_ $$2doi$$a10.1002/mdc3.13232 000892509 0247_ $$2Handle$$a2128/28691 000892509 0247_ $$2altmetric$$aaltmetric:104828514 000892509 0247_ $$2pmid$$apmid:34307758 000892509 0247_ $$2WOS$$aWOS:000647076200001 000892509 037__ $$aFZJ-2021-02118 000892509 041__ $$aEnglish 000892509 082__ $$a610 000892509 1001_ $$0P:(DE-HGF)0$$aPetry-Schmelzer, Jan Niklas$$b0$$eCorresponding author 000892509 245__ $$aVPS13D : One Family, Same Mutations, Two Phenotypes 000892509 260__ $$aNew York, NY$$bWiley$$c2021 000892509 3367_ $$2DRIVER$$aarticle 000892509 3367_ $$2DataCite$$aOutput Types/Journal article 000892509 3367_ $$0PUB:(DE-HGF)16$$2PUB:(DE-HGF)$$aJournal Article$$bjournal$$mjournal$$s1632473974_765 000892509 3367_ $$2BibTeX$$aARTICLE 000892509 3367_ $$2ORCID$$aJOURNAL_ARTICLE 000892509 3367_ $$00$$2EndNote$$aJournal Article 000892509 520__ $$aHere, we report two siblings with compound heterozygous variants in VPS13D but a considerable divergent phenotype highlighting the difficulties of phenotype–genotype correlation in this rare disease.A 19-year-old patient presented to our outpatient department with a five-year history of progressive gait impairment. Motor and cognitive development during childhood was described as normal. The patient visited a regular school and was able to participate in physical education until the onset of symptoms. There were no known neurologic diseases in the Caucasian family, consisting of five sisters (II.1–3, II.5, and II.6), and the patient (II.4). However, one younger sister (II.6) of the patient suffered from developmental delay and intellectual disability without further physical impairment, and the oldest sister (II.1) living separate from the family was supposed to have “gait problems”. 000892509 536__ $$0G:(DE-HGF)POF4-525$$a525 - Decoding Brain Organization and Dysfunction (POF4-525)$$cPOF4-525$$fPOF IV$$x0 000892509 588__ $$aDataset connected to DataCite 000892509 7001_ $$0P:(DE-HGF)0$$aKeller, Natalie$$b1 000892509 7001_ $$0P:(DE-HGF)0$$aKarakaya, Mert$$b2 000892509 7001_ $$0P:(DE-HGF)0$$aWirth, Brunhilde$$b3 000892509 7001_ $$0P:(DE-Juel1)131720$$aFink, Gereon R.$$b4 000892509 7001_ $$0P:(DE-HGF)0$$aWunderlich, Gilbert$$b5 000892509 773__ $$0PERI:(DE-600)2772809-2$$a10.1002/mdc3.13232$$gp. mdc3.13232$$n5$$p803-806$$tMovement disorders clinical practice$$v8$$x2330-1619$$y2021 000892509 8564_ $$uhttps://juser.fz-juelich.de/record/892509/files/mdc3.13232-1.pdf 000892509 8564_ $$uhttps://juser.fz-juelich.de/record/892509/files/Petry-Schmelzer_Movement%20Disord_One%20familiy%2C%20same...post%20print.pdf$$yPublished on 2021-04-27. Available in OpenAccess from 2022-04-27. 000892509 909CO $$ooai:juser.fz-juelich.de:892509$$pdnbdelivery$$pdriver$$pVDB$$popen_access$$popenaire 000892509 9101_ $$0I:(DE-588b)5008462-8$$6P:(DE-Juel1)131720$$aForschungszentrum Jülich$$b4$$kFZJ 000892509 9131_ $$0G:(DE-HGF)POF4-525$$1G:(DE-HGF)POF4-520$$2G:(DE-HGF)POF4-500$$3G:(DE-HGF)POF4$$4G:(DE-HGF)POF$$aDE-HGF$$bKey Technologies$$lNatural, Artificial and Cognitive Information Processing$$vDecoding Brain Organization and Dysfunction$$x0 000892509 9130_ $$0G:(DE-HGF)POF3-572$$1G:(DE-HGF)POF3-570$$2G:(DE-HGF)POF3-500$$3G:(DE-HGF)POF3$$4G:(DE-HGF)POF$$aDE-HGF$$bKey Technologies$$lDecoding the Human Brain$$v(Dys-)function and Plasticity$$x0 000892509 9141_ $$y2021 000892509 915__ $$0StatID:(DE-HGF)0200$$2StatID$$aDBCoverage$$bSCOPUS$$d2020-08-21 000892509 915__ $$0StatID:(DE-HGF)0600$$2StatID$$aDBCoverage$$bEbsco Academic Search$$d2020-08-21 000892509 915__ $$0StatID:(DE-HGF)0530$$2StatID$$aEmbargoed OpenAccess 000892509 915__ $$0StatID:(DE-HGF)0112$$2StatID$$aWoS$$bEmerging Sources Citation Index$$d2020-08-21 000892509 915__ $$0StatID:(DE-HGF)3001$$2StatID$$aDEAL Wiley$$d2020-08-21$$wger 000892509 915__ $$0StatID:(DE-HGF)0150$$2StatID$$aDBCoverage$$bWeb of Science Core Collection$$d2020-08-21 000892509 915__ $$0StatID:(DE-HGF)0030$$2StatID$$aPeer Review$$bASC$$d2020-08-21 000892509 915__ $$0StatID:(DE-HGF)0300$$2StatID$$aDBCoverage$$bMedline$$d2020-08-21 000892509 915__ $$0StatID:(DE-HGF)0320$$2StatID$$aDBCoverage$$bPubMed Central$$d2020-08-21 000892509 915__ $$0StatID:(DE-HGF)0199$$2StatID$$aDBCoverage$$bClarivate Analytics Master Journal List$$d2020-08-21 000892509 920__ $$lyes 000892509 9201_ $$0I:(DE-Juel1)INM-3-20090406$$kINM-3$$lKognitive Neurowissenschaften$$x0 000892509 980__ $$ajournal 000892509 980__ $$aVDB 000892509 980__ $$aUNRESTRICTED 000892509 980__ $$aI:(DE-Juel1)INM-3-20090406 000892509 9801_ $$aFullTexts