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The marketing authorization for Glybera clearly represents a milestone in the development of gene therapy as an accessible therapeutic option for familial lipoprotein lipase deficiency (LPLD) patients. Glybera is a recombinant __________ for direct intramuscular injection.
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In March 2017, Yescarta (axicabtagene ciloleucel), a ________-directed genetically modified autologous T cell immunotherapy, was approved by FDA.
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.
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.
_____________ 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.
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