{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,20]],"date-time":"2026-03-20T04:30:34Z","timestamp":1773981034404,"version":"3.50.1"},"reference-count":9,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2018,12,7]],"date-time":"2018-12-07T00:00:00Z","timestamp":1544140800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Transplantation"],"published-print":{"date-parts":[[2019,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Danon disease (DD) is an X\u2010linked dominant disorder caused by a mutation in the lysosomal\u2010associated membrane protein\u20102 (LAMP\u20102) gene coding for the LAMP\u20102 protein. We report two cases of successful heart transplantation (HT) in adolescent brothers with DD, including one who was bridged to HT for 34\u00a0days with a HeartWare left ventricular assist device. In both patients, the post\u2010transplant course was complicated by profound skeletal muscle weakness that resolved with corticosteroid withdrawal. These cases highlight that both HT and ventricular assist device support are feasible in patients with DD. Corticosteroid use may exacerbate skeletal myopathy, and therefore, steroid minimization may be warranted whenever possible.<\/jats:p>","DOI":"10.1111\/petr.13335","type":"journal-article","created":{"date-parts":[[2018,12,8]],"date-time":"2018-12-08T07:40:50Z","timestamp":1544254850000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":10,"title":["Heart transplantation in two adolescents with Danon disease"],"prefix":"10.1111","volume":"23","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2447-3713","authenticated-orcid":false,"given":"Daniel","family":"Oren","sequence":"first","affiliation":[{"name":"Heart and Vascular Center Semmelweis University Budapest Hungary"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Peter","family":"Chau","sequence":"additional","affiliation":[{"name":"Department of Pediatrics (Cardiology) Loma Linda Medical Center Loma Linda California"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Melanie","family":"Manning","sequence":"additional","affiliation":[{"name":"Department of Pediatrics (Medical Genetics) and Pathology, Stanford University Medical Center Stanford University Stanford California"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Joann","family":"Kwong","sequence":"additional","affiliation":[{"name":"Rehabilitation Services Lucile Packard Children\u2019s Hospital Stanford California"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0484-5281","authenticated-orcid":false,"given":"Beth D.","family":"Kaufman","sequence":"additional","affiliation":[{"name":"Department of Pediatrics (Cardiology), Stanford University Medical Center Stanford University Stanford California"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Katsuhide","family":"Maeda","sequence":"additional","affiliation":[{"name":"Department of Cardiothoracic Surgery Stanford University Medical Center Stanford California"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"David N.","family":"Rosenthal","sequence":"additional","affiliation":[{"name":"Department of Pediatrics (Cardiology), Stanford University Medical Center Stanford University Stanford California"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0818-3150","authenticated-orcid":false,"given":"Seth A.","family":"Hollander","sequence":"additional","affiliation":[{"name":"Department of Pediatrics (Cardiology), Stanford University Medical Center Stanford University Stanford California"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2018,12,7]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/35022604"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCHEARTFAILURE.114.001105"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2009.371"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1097\/GIM.0b013e31820ad795"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.amjcard.2011.09.024"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.58.12.1773"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.athoracsur.2016.07.022"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.healun.2017.10.027"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.2353\/ajpath.2006.050646"}],"container-title":["Pediatric Transplantation"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1111%2Fpetr.13335","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/petr.13335","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1111\/petr.13335","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/petr.13335","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,14]],"date-time":"2023-09-14T03:39:49Z","timestamp":1694662789000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/petr.13335"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,12,7]]},"references-count":9,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2019,3]]}},"alternative-id":["10.1111\/petr.13335"],"URL":"https:\/\/doi.org\/10.1111\/petr.13335","archive":["Portico"],"relation":{},"ISSN":["1397-3142","1399-3046"],"issn-type":[{"value":"1397-3142","type":"print"},{"value":"1399-3046","type":"electronic"}],"subject":[],"published":{"date-parts":[[2018,12,7]]},"assertion":[{"value":"2018-08-08","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2018-11-08","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2018-12-07","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e13335"}}