HAVING A GO AT SPINAL MUSCULAR ATROPHY WITH SPINRAZA
DOI:
https://doi.org/10.22159/ajpcr.2017.v10i6.17502Keywords:
Spinraza, Spinal muscualr atrophy, Antisense oligonucleotide, Adverse effects, Endear trialAbstract
Spinal muscular atrophy (SMA), a neurological condition which is genetically mediated is the second most common infantile disease causing morbidity and mortality next to cystic fibrosis. It is of five different types with each type having different severity outcomes. For almost three decades, only supportive measures were advocated in the treatment of SMA. Recently, Biogen's Spinraza came out as the first disease modifying therapy to treat infantile as well as adult SMA. This review throws light on the pharmacological aspects of the drug; its approval by Food and Drug Administration and various completed clinical trials as well ongoing clinical trials.
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