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FUNDING The present research has not received specific financial support from either the private or public sector

Concomitant administration of Carnitene with drugs that induce hypocarnitinemia due to increased renal carnitine loss (valproic acid, prodrugs containing pivalic acid, cephalosporins, cisplatin, carboplatin and ifosfamide) may increase the risk of hypocarnitinemia

Results are provided as mean changes in SGRQ Total score and as response analysis (4-point improvement [responder], no change, and 4-point worsening in Total score) using analysis of covariance or logistic regression, as applicable

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BTHAL is an autosomal recessive disorder presenting with high frequencies in populations from the Mediterranean area