{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T05:19:35Z","timestamp":1788239975088,"version":"build-2803163510"},"reference-count":28,"publisher":"Wiley","license":[{"start":{"date-parts":[[2026,8,31]],"date-time":"2026-08-31T00:00:00Z","timestamp":1788134400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,8,31]],"date-time":"2026-08-31T00:00:00Z","timestamp":1788134400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100008484","name":"BioMarin Pharmaceutical","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008484","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["American J of Med Genetics Pt A"],"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>\n                    Achondroplasia is a skeletal dysplasia associated with severe short stature and multisystem complications due to a gain\u2010of\u2010function pathogenic variant in the\n                    <jats:italic>FGFR3<\/jats:italic>\n                    gene. Vosoritide, a C\u2010type natriuretic peptide analog, has demonstrated efficacy in clinical trials. The objective of this study was to describe the effectiveness, safety, treatment continuity, and caregiver\u2010reported outcomes of vosoritide in routine practice across heterogeneous health systems in Latin America in a real\u2010world context. We conducted a retrospective, multicenter, multinational cohort study including children with molecularly confirmed achondroplasia treated with vosoritide in centers from Argentina, Colombia, Uruguay, Mexico, and Chile. Eligible patients had \u2265\u20096\u2009months of treatment and at least two anthropometric evaluations. Height, annualized growth velocity (AGV), body proportion measures, treatment interruptions, adverse events, and parent\u2010reported outcomes were analyzed. Fifty\u2010two patients (mean age 5.9\u2009years at treatment initiation) were included, with a mean treatment duration of 539\u2009days and high adherence (93.9% time without interruptions). Height\n                    <jats:italic>Z<\/jats:italic>\n                    \u2010scores increased significantly from 6 to 36\u2009months (0.33 to 1.58;\n                    <jats:italic>p<\/jats:italic>\n                    \u2009&lt;\u20090.001 to\n                    <jats:italic>p<\/jats:italic>\n                    \u2009&lt;\u20090.05). The most significant impact in AVG is observed during the first year of treatment with vosoritide, followed by a subsequent stabilization, reaching growth rates comparable to those of the healthy population. Increases were consistent across sexes and age groups. All patients were in a higher percentile range for AGV during treatment compared to before treatment, in some cases exceeding the 95th percentile, when compared to reference curves. Body proportion indices remained stable. All patients demonstrated gains in linear growth, although the magnitude of increase varied across individuals. Adverse events were frequent but mild, mainly injection\u2010site reactions; no severe adverse events or treatment discontinuations due to safety issues occurred. Parent\u2010reported outcomes were highly favorable, with 98% of caregivers perceiving positive changes in their children's overall well\u2010being, daily functioning, or quality of life, and all caregivers expressing willingness to recommend the treatment. In real\u2010world clinical practice across Latin America, vosoritide demonstrated rapid and sustained gains in linear growth, favorable tolerability, high adherence, and meaningful benefits as perceived by caregivers. These results reinforce the generalizability of vosoritide therapy in heterogeneous healthcare systems and highlight the relevance of real\u2010world evidence for informing treatment decisions in rare diseases.\n                  <\/jats:p>","DOI":"10.1002\/ajmg.a.70254","type":"journal-article","created":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T05:03:47Z","timestamp":1788239027000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Real\u2010World Evidence on the Effectiveness and Safety of Vosoritide in Latin American Patients With Achondroplasia (\n                    <scp>EVOLAC<\/scp>\n                    )"],"prefix":"10.1002","author":[{"ORCID":"https:\/\/orcid.org\/0009-0000-1777-8124","authenticated-orcid":false,"given":"Julieta","family":"De Victor","sequence":"first","affiliation":[{"name":"Hospital Interzonal General de Agudos Eva Per\u00f3n  San Mart\u00edn Buenos Aires Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3094-6855","authenticated-orcid":false,"given":"Eduardo D.","family":"Gil","sequence":"additional","affiliation":[{"name":"Hospital Interzonal Especializado Materno Infantil \u201cVictorio Tetamanti\u201d  Mar del Plata Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0000-8337-219X","authenticated-orcid":false,"given":"Florencia","family":"Pabletich","sequence":"additional","affiliation":[{"name":"Hospital Privado Universitario de C\u00f3rdoba  C\u00f3rdoba Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0007-4430-5518","authenticated-orcid":false,"given":"Roc\u00edo Rabosto","family":"Mole\u00f3n","sequence":"additional","affiliation":[{"name":"Private Practice  Santa Fe Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carolina A.","family":"Dellamea","sequence":"additional","affiliation":[{"name":"Hospital Pedi\u00e1trico \u201cAvelino Castel\u00e1n\u201d  Resistencia Chaco Argentina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3874-3964","authenticated-orcid":false,"given":"M\u00f3nica","family":"Fern\u00e1ndez","sequence":"additional","affiliation":[{"name":"Fundaci\u00f3n Cardioinfantil  Bogot\u00e1 Colombia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3532-2002","authenticated-orcid":false,"given":"Norma C.","family":"Serrano","sequence":"additional","affiliation":[{"name":"Fundaci\u00f3n Cardiovascular de Colombia \u2013 Hospital Internacional de Colombia  Bucaramanga 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Ojeda","sequence":"additional","affiliation":[{"name":"CRIT Occidente, Fundaci\u00f3n Telet\u00f3n  Guadalajara Jalisco Mexico"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8330-9710","authenticated-orcid":false,"given":"Beatriz E.","family":"De la Fuente\u2010Cortez","sequence":"additional","affiliation":[{"name":"Hospital Universitario \u201cDr. Jos\u00e9 Eleuterio Gonz\u00e1lez\u201d, UANL  Monterrey Mexico"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nancy","family":"Unanue","sequence":"additional","affiliation":[{"name":"IDIMI \u2013 Universidad de Chile  Santiago Chile"},{"name":"Hospital Cl\u00ednico San Borja Arriar\u00e1n  Santiago Chile"},{"name":"Cl\u00ednica Las Condes  Santiago Chile"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6680-793X","authenticated-orcid":false,"given":"Mar\u00eda 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