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330 SMA is a genetic neuromuscular disease caused by the deletion or mutation of the survival motor neuron 1 (SMN1) gene, leading to the loss of alpha motor neurons and progressive muscle atrophy
After one or two infusions patients usually have substantially higher iron levels for prolonged periods of time
The FDA defines a prognostic biomarker as a measured characteristic that reflects a patients degree of risk of disease occurrence or progression that is independent of treatment while a predictive biomarker categorizes patients by their likelihood to respond favorably or adversely to a particular treatment
falciparum IC 50 values (2 M)
ROC analysis of adduct levels in serum from patients known to have APAP hepatotoxicity showed that an APAP protein adduct level 1.1 nmol/mL had a high sensitivity and specificity (96.8 and 95.0 %, respectively) for patients with APAP overdose and ALT levels above 1000 IU/L
Robbins TW (2005)
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