题目内容

On August 30, 2017, the US FDA approved Kymriah (tisagenlecleucel, Novartis)for the treatment of patients aged ≤25 years with B-cell precursor ______________________ that is refractory, in second relapse, or later relapse.

A. acute myelogenous (or myeloid) leukemia (AML)
B. acute lymphocytic (or lymphoblastic) leukemia (ALL)
C. chronic myelogenous (or myeloid) leukemia (CML)
D. chronic lymphocytic (or lymphoblastic) leukemia (CLL)

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On 24 May 2019, Zolgensma (onasemnogene abeparvovec-xioi, AVXS-101) was approved by the FDA indicated for the treatment of pediatric patients less than ___ years of age with spinal muscular atrophy (SMA) with biallelic mutations in the survival motor neuron 1 (SMN1) gene.

A. 1
B. 2
C. 3
D. 5

_____________ 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

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