Open AccessHAVING A GO AT SPINAL MUSCULAR ATROPHY WITH SPINRAZA
Author(s) -
O Balaji,
Amita Priya,
Sereen Rose Thomson,
Navin Patil
Publication year - 2017
Publication title -
asian journal of pharmaceutical and clinical research
Language(s) - English
Resource type - Journals
eISSN - 2455-3891
pISSN - 0974-2441
DOI - 10.22159/ajpcr.2017.v10i6.17502
Subject(s) - spinal muscular atrophy , sma* , medicine , food and drug administration , clinical trial , disease , cystic fibrosis , atrophy , pediatrics , physical medicine and rehabilitation , intensive care medicine , pathology , pharmacology , mathematics , combinatorics
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.