{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,3]],"date-time":"2026-10-03T10:00:10Z","timestamp":1791021610496,"version":"4.1.0"},"reference-count":22,"publisher":"Georg Thieme Verlag KG","issue":"08","license":[{"start":{"date-parts":[[2025,8,20]],"date-time":"2025-08-20T00:00:00Z","timestamp":1755648000000},"content-version":"vor","delay-in-days":19,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Arq Neuropsiquiatr"],"published-print":{"date-parts":[[2025,8]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Charcot-Marie-Tooth disease (CMT) is a progressive neurological disorder that typically manifests in early childhood. The natural progression of CMT in Brazilian pediatric and adolescent populations remains unknown.<\/jats:p>\n                  <jats:p>To evaluate the natural disease progression in Brazilian children and adolescents with CMT using the Charcot-Marie-Tooth Pediatric Scale (CMTPedS).<\/jats:p>\n                  <jats:p>A longitudinal observational study was conducted to assess disease progression over a 2-year period through 3 evaluations, spaced 1 year apart, in children and adolescents aged 5 to 18 years with a confirmed CMT diagnosis. Height, weight, body mass index (BMI), fat-free mass, and functionality (assessed via the CMTPedS) were evaluated in each of the three sessions.<\/jats:p>\n                  <jats:p>We included 30 participants of both sexes with a mean age of 11.1\u2009\u00b1\u20093.2 years. Significant increases in height, weight, and BMI were observed, alongside a decline in the percentage of lean body mass across evaluations. The total score on the CMTPedS increased by 4.5 points throughout 2 years, indicating disease progression, with notable deterioration in functional dexterity, pinprick and vibration sensations, and gait. Significant progression was evident within 1 year, with an average annual deterioration of 2.25 points. The subgroup with CMT type 1A (CMT1A) presented an increase of 4 points in the total score, corresponding to an average annual progression of 2 points. Unlike the overall CMT group, the CMT1 subgroup did not exhibit a decline in the pinprick sensation score.<\/jats:p>\n                  <jats:p>Children and adolescents with CMT exhibit significant disease progression within 2 years, with measurable deterioration observed as early as 1 year. The CMTPedS is a reliable tool to monitor CMT progression in this population.<\/jats:p>","DOI":"10.1055\/s-0045-1811174","type":"journal-article","created":{"date-parts":[[2025,8,20]],"date-time":"2025-08-20T23:41:01Z","timestamp":1755733261000},"page":"001-008","source":"Crossref","is-referenced-by-count":2,"title":["Longitudinal assessment of natural disease progression in Brazilian children and adolescents with Charcot-Marie-Tooth disease"],"prefix":"10.1055","volume":"83","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1709-3747","authenticated-orcid":false,"given":"Bruno Alvarenga","family":"Soares","sequence":"additional","affiliation":[{"name":"Universidade de S\u00e3o Paulo, Faculdade de Medicina de Ribeir\u00e3o Preto, Ribeir\u00e3o Preto SP, Brazil."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0000-1668-8821","authenticated-orcid":false,"given":"Camila Fernanda de","family":"Freitas","sequence":"additional","affiliation":[{"name":"Universidade de S\u00e3o Paulo, Faculdade de Medicina de Ribeir\u00e3o Preto, Ribeir\u00e3o Preto SP, Brazil."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5870-261X","authenticated-orcid":false,"given":"Juliana","family":"Cardoso","sequence":"additional","affiliation":[{"name":"Universidade de S\u00e3o Paulo, Faculdade de Medicina de Ribeir\u00e3o Preto, Ribeir\u00e3o Preto SP, Brazil."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5850-9940","authenticated-orcid":false,"given":"Cyntia Rogean de Jesus Alves de","family":"Baptista","sequence":"additional","affiliation":[{"name":"Universidade de S\u00e3o Paulo, Faculdade de Medicina de Ribeir\u00e3o Preto, Ribeir\u00e3o Preto SP, Brazil."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4589-2749","authenticated-orcid":false,"suffix":"Junior","given":"Wilson","family":"Marques","sequence":"additional","affiliation":[{"name":"Universidade de S\u00e3o Paulo, Faculdade de Medicina de Ribeir\u00e3o Preto, Ribeir\u00e3o Preto SP, Brazil."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4181-0718","authenticated-orcid":false,"given":"Ana Claudia","family":"Mattiello-Sverzut","sequence":"additional","affiliation":[{"name":"Universidade de S\u00e3o Paulo, Faculdade de Medicina de Ribeir\u00e3o Preto, Ribeir\u00e3o Preto SP, Brazil."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"194","published-online":{"date-parts":[[2025,8,20]]},"reference":[{"issue":"05","key":"ref1","doi-asserted-by":"publisher","first-page":"471","DOI":"10.1097\/WCO.0000000000000474","article-title":"New developments in 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