Retrospective evaluation of patients with X-linked adrenoleukodystrophy with a wide range of clinical presentations: a single center experience
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2021
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Abstract
Patients with X-ALD should be carefully followed up for cerebral findings and progression, since there is no genotype-phenotype correlation, and the clinical course cannot be predicted by family history. HSCT is the only available treatment option for patients with neurological deterioration.
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| Authors | Olgac A;Kasapkara ÇS;Derinkuyu B;Yüksel D;Çetinkaya S;Aksoy A;Ceylaner S;Güleray N;Yeşilipek A;Aydın Hİ;Orgun LT;Kılıç M;; |
| Journal | journal of pediatric endocrinology & metabolism : jpem |
| Year | 2021 |
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