_____________ is the first therapeutic treatment approved to treat the rare and fatal spinal muscular atrophy (SMA) disease. SMA is a severe neuromuscular disease that causes extreme muscle weakness and paralysis caused by a genetic defect in the SMN1 gene.
A. Luxturna
B. Kymriah
C. Zolgensma
D. Yescarta
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Spinal muscular atrophy (SMA)is diagnosed in approximately one out of every _________ live births and is the leading genetic cause of infant mortality.
A. 8,000
B. 10,000
C. 12,000
D. 15,000
Zolgensma is supplied as a suspension for intravenous administration. It is for single-dose intravenous infusion only. The recommended dosage of Zolgensma is 1.1×_______ vector genomes (vg) per kg of body weight. Zolgensma is administered as an intravenous infusion over 60 minutes.
A. 10^10
B. 10^12
C. 10^14
D. 10^16
Zolgensma is provided in a kit containing 2 to 9 vials, as a combination of 2 vial fill volumes (either 5.5 ml or 8.3 ml). All vials have a nominal concentration of 2.0×_______ vector genomes (vg) per ml. Each vial contains an extractable volume of not less than either 5.5 ml or 8.3 ml.
A. 10^12
B. 10^13
C. 10^14
D. 10^16
Themostcommonadversereactions(incidence≥5%) of Zolgensma are elevatedaminotransferasesand___________.
A. nausea
B. vomiting
C. fever
D. headache