In March 2017, Yescarta (axicabtagene ciloleucel), a ________-directed genetically modified autologous T cell immunotherapy, was approved by FDA.
A. CD15
B. CD19
CD29
D. CD44
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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)
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