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The primary outcome measure was prolongation of the ability to walk.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results:<\/jats:title><jats:p>One patient lost ambulation at age 10. Four patients, aged 16 to 18 were fully ambulant, and 3 of them could still climb stairs. Respiratory function was moderately reduced in 2. Left ventricular ejection fraction was &gt; 45%. Short stature and delayed puberty were the most relevant side effects. Although the negative impact of corticosteroid treatment on growth rate remained their major concern, parents and patients stated that they preferred corticosteroid therapy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions:<\/jats:title><jats:p>Long\u2010term corticosteroid treatment is effective in prolonging function but not in recovering lost function, and its early use seems appropriate. 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