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Having a go at spinal muscular atrophy with spinraza

Balaji, O and Priya, Amita and Thomson, Sereen Rose and Patil, Navin (2017) Having a go at spinal muscular atrophy with spinraza. Asian Journal of Pharmaceutical and Clinical Research, 10 (6). pp. 16-18. ISSN 0974-2441

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Abstract

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.

Item Type: Article
Uncontrolled Keywords: Spinraza; spinal muscualr atrophy; antisense oligonucleotide; adverse effects; endear trial.
Subjects: Medicine > KMC Manipal > Pharmacology
Depositing User: KMC Library
Date Deposited: 19 Jun 2017 09:19
Last Modified: 19 Jun 2017 09:19
URI: http://eprints.manipal.edu/id/eprint/149089

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