{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,11]],"date-time":"2026-10-11T21:08:27Z","timestamp":1791752907200,"version":"4.3.4"},"reference-count":39,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2007,2,19]],"date-time":"2007-02-19T00:00:00Z","timestamp":1171843200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Clinical Genetics"],"published-print":{"date-parts":[[2007,3]]},"abstract":"<jats:p>The effect of enzyme replacement therapy (ERT) on bone crisis and bone pain was investigated in patients with Gaucher disease (GD) type 1 followed over 4 years. Data from the International Collaborative Gaucher Group Gaucher Registry were used. Only patients with bone crisis and\/or bone pain data for 1 year prior to ERT, and for each of 3 years after the start of ERT, were included. Bone crises were reported in 17% of patients during the year before starting ERT. The frequencies of bone crises decreased to 5%, &lt;1% and 3% for 1, 2, and 3 years after initiation of treatment, respectively (p &lt; 0.0001). Bone pain followed a similar pattern of response. Bone pain was reported in 49% of patients the year before treatment and decreased to 30% in the first year, 29% in the second year, and 30% in the third year of ERT (p &lt; 0.0001). ERT is associated with a reduction in bone crisis and bone pain in patients with GD type 1 . This study shows that significant improvements in symptoms of skeletal disease are achievable clinical outcomes and treatment goals in GD type 1.<\/jats:p>","DOI":"10.1111\/j.1399-0004.2007.00769.x","type":"journal-article","created":{"date-parts":[[2007,2,19]],"date-time":"2007-02-19T12:49:04Z","timestamp":1171889344000},"page":"205-211","source":"Crossref","is-referenced-by-count":111,"title":["The effect of enzyme replacement therapy on bone crisis and bone pain in patients with type 1 Gaucher disease"],"prefix":"10.1111","volume":"71","author":[{"given":"J","family":"Charrow","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"B","family":"Dulisse","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"GA","family":"Grabowski","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"NJ","family":"Weinreb","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2007,2,19]]},"reference":[{"key":"e_1_2_6_2_2","first-page":"3635","volume-title":"The metabolic and molecular bases of inherited diseases","author":"Beutler E","year":"1991"},{"key":"e_1_2_6_3_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0950-3536(97)80033-9"},{"key":"e_1_2_6_4_2","doi-asserted-by":"publisher","DOI":"10.1001\/archinte.160.18.2835"},{"key":"e_1_2_6_5_2","doi-asserted-by":"publisher","DOI":"10.1097\/00005792-198509000-00003"},{"key":"e_1_2_6_6_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0022-3476(96)70203-2"},{"key":"e_1_2_6_7_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00431-003-1362-0"},{"key":"e_1_2_6_8_2","doi-asserted-by":"publisher","DOI":"10.1148\/radiology.170.1.2909087"},{"key":"e_1_2_6_9_2","doi-asserted-by":"publisher","DOI":"10.2106\/00004623-198668090-00011"},{"key":"e_1_2_6_10_2","first-page":"33","article-title":"Phenotypic manifestations of Gaucher disease: clinical features in 48 biochemically verified type 1 patients and comment on type 2 patients","volume":"95","author":"Kolodny EH","year":"1982","journal-title":"Prog Clin Biol Res"},{"key":"e_1_2_6_11_2","doi-asserted-by":"publisher","DOI":"10.1001\/archinte.158.16.1754"},{"key":"e_1_2_6_12_2","doi-asserted-by":"publisher","DOI":"10.1023\/A:1008814105603"},{"key":"e_1_2_6_13_2","doi-asserted-by":"publisher","DOI":"10.1007\/s11136-004-0794-y"},{"key":"e_1_2_6_14_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2003.10.067"},{"key":"e_1_2_6_15_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0268-960X(98)90023-6"},{"key":"e_1_2_6_16_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199105233242104"},{"key":"e_1_2_6_17_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0002-9343(02)01150-6"},{"key":"e_1_2_6_18_2","doi-asserted-by":"publisher","DOI":"10.1542\/peds.96.4.629"},{"key":"e_1_2_6_19_2","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-005-0032-y"},{"key":"e_1_2_6_20_2","doi-asserted-by":"publisher","DOI":"10.1006\/bcmd.1996.0016"},{"key":"e_1_2_6_21_2","first-page":"25","article-title":"First long\u2010term results of imiglucerase therapy of type 1 Gaucher disease","volume":"3","author":"Niederau C","year":"1998","journal-title":"Eur J Med Res"},{"key":"e_1_2_6_22_2","doi-asserted-by":"publisher","DOI":"10.1023\/A:1008878425167"},{"key":"e_1_2_6_23_2","doi-asserted-by":"publisher","DOI":"10.1034\/j.1600-0609.2003.00047.x"},{"key":"e_1_2_6_24_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2005-12-5072"},{"key":"e_1_2_6_25_2","doi-asserted-by":"publisher","DOI":"10.1006\/bcmd.1998.0195"},{"key":"e_1_2_6_26_2","doi-asserted-by":"publisher","DOI":"10.1111\/j.1442-200X.1996.tb03482.x"},{"key":"e_1_2_6_27_2","doi-asserted-by":"publisher","DOI":"10.1023\/A:1005451300167"},{"key":"e_1_2_6_28_2","first-page":"749","article-title":"Enzyme replacement therapy in Gaucher disease: monitoring visceral and bone changes with MRI","volume":"144","author":"Toth J","year":"2003","journal-title":"Orv Hetil"},{"key":"e_1_2_6_29_2","first-page":"595","article-title":"Osteonecrosis in a patient with Gaucher\u2019s disease treated with enzyme replacement","volume":"5","author":"Lebel E","year":"2003","journal-title":"Isr Med Assoc J"},{"key":"e_1_2_6_30_2","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.2830470325"},{"key":"e_1_2_6_31_2","doi-asserted-by":"publisher","DOI":"10.1259\/bjr.75.suppl_1.750025"},{"key":"e_1_2_6_32_2","unstructured":"International Collaborative Gaucher Group Registry. 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