{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,23]],"date-time":"2026-10-23T22:50:21Z","timestamp":1792795821738,"version":"4.5.0"},"reference-count":20,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2019,6,30]],"date-time":"2019-06-30T00:00:00Z","timestamp":1561852800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"name":"Friedreich Ataxia Research Allaince"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2019,11]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>In this study we assessed the effect of methylprednisolone on safety, tolerability, and ability in Friedreich ataxia (FRDA).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>The study was an open\u2010label trial of pulse methylprednisolone on 11 participants with FRDA. All participants followed a 28\u2010day treatment cycle, repeated 7 times. Patients were assessed with the timed 25\u2010foot walk (T25FW), 1\u2010minute walk (1MW), the Friedreich Ataxia Rating Scale (FARS), and the 9\u2010hole peg test (9HPT). Efficacy was tested by comparing baseline and week 26 visits, separated into adult and pediatric groups.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      In comparisons of participants\u2019 baseline and week 26 visits, only the pediatric cohort's 1MW score showed change (\n                      <jats:italic>P<\/jats:italic>\n                      \u2009&lt;\u20090.05). The T25FW, the primary outcome measure, did not change significantly.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>Pediatric participants improved their gait distance in the 1MW, but did not significantly improve in other measures in this overall negative study. Methylprednisolone was generally well tolerated, suggesting that it may be useful for ambulatory children with FRDA if benefit is found with further study.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mus.26610","type":"journal-article","created":{"date-parts":[[2019,6,17]],"date-time":"2019-06-17T12:20:26Z","timestamp":1560774026000},"page":"571-575","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":15,"title":["Open\u2010label pilot study of oral methylprednisolone for the treatment of patients with friedreich ataxia"],"prefix":"10.1002","volume":"60","author":[{"given":"Maya","family":"Patel","sequence":"first","affiliation":[{"name":"Division of Neurology, Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Boulevard  Philadelphia Pennsylvania 19104\u20104318 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kimberly","family":"Schadt","sequence":"additional","affiliation":[{"name":"Division of Neurology, Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Boulevard  Philadelphia Pennsylvania 19104\u20104318 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ashley","family":"McCormick","sequence":"additional","affiliation":[{"name":"Division of Neurology, Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Boulevard  Philadelphia Pennsylvania 19104\u20104318 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Charles","family":"Isaacs","sequence":"additional","affiliation":[{"name":"Division of Neurology, Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Boulevard  Philadelphia Pennsylvania 19104\u20104318 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yi Na","family":"Dong","sequence":"additional","affiliation":[{"name":"Division of Neurology, Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Boulevard  Philadelphia Pennsylvania 19104\u20104318 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David R.","family":"Lynch","sequence":"additional","affiliation":[{"name":"Division of Neurology, Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Boulevard  Philadelphia Pennsylvania 19104\u20104318 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2019,6,30]]},"reference":[{"key":"e_1_2_6_2_1","doi-asserted-by":"publisher","DOI":"10.1001\/archneur.65.10.1296"},{"key":"e_1_2_6_3_1","doi-asserted-by":"publisher","DOI":"10.1001\/archneur.59.5.743"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.1177\/0883073812448230"},{"key":"e_1_2_6_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.24595"},{"key":"e_1_2_6_6_1","doi-asserted-by":"publisher","DOI":"10.3389\/fphar.2014.00130"},{"key":"e_1_2_6_7_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.96.20.11492"},{"key":"e_1_2_6_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.10419"},{"key":"e_1_2_6_9_1","doi-asserted-by":"publisher","DOI":"10.1186\/s12883-016-0526-2"},{"key":"e_1_2_6_10_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000002337"},{"key":"e_1_2_6_11_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddv024"},{"key":"e_1_2_6_12_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pgen.1000812"},{"key":"e_1_2_6_13_1","doi-asserted-by":"publisher","DOI":"10.1212\/01.WNL.0000156802.15466.79"},{"key":"e_1_2_6_14_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.22912"},{"key":"e_1_2_6_15_1","doi-asserted-by":"publisher","DOI":"10.1212\/01.wnl.0000218155.46739.90"},{"key":"e_1_2_6_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.20449"},{"key":"e_1_2_6_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jns.2008.05.009"},{"key":"e_1_2_6_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2010.07.001"},{"key":"e_1_2_6_19_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.mito.2010.12.003"},{"key":"e_1_2_6_20_1","doi-asserted-by":"publisher","DOI":"10.1001\/archneurol.2010.168"},{"key":"e_1_2_6_21_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12311-014-0568-1"}],"container-title":["Muscle &amp; 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