HAVING A GO AT SPINAL MUSCULAR ATROPHY WITH SPINRAZA


Balaji O, Amita D, Sereen Rt, Navin Ap

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.


Keywords


Spinraza, Spinal muscualr atrophy, Antisense oligonucleotide, Adverse effects, Endear trial.

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References


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About this article

Title

HAVING A GO AT SPINAL MUSCULAR ATROPHY WITH SPINRAZA

Keywords

Spinraza, Spinal muscualr atrophy, Antisense oligonucleotide, Adverse effects, Endear trial.

DOI

10.22159/ajpcr.2017.v10i6.17502

Date

01-06-2017

Additional Links

Manuscript Submission

Journal

Asian Journal of Pharmaceutical and Clinical Research
Vol 10 Issue 6 June 2017 Page: 16-18

Print ISSN

0974-2441

Online ISSN

2455-3891

Statistics

167 Views | 154 Downloads

Authors & Affiliations

Balaji O
Department of Pharmacology, Kasturba Medical College, Manipal, Karnataka, India.
India

Amita D
Department of Pharmacology, Kasturba Medical College, Manipal, Karnataka, India.
India

Sereen Rt
Department of Pharmacology, Kasturba Medical College, Manipal, Karnataka, India.
India

Navin Ap
Department of Pharmacology, Kasturba Medical College, Manipal, Karnataka, India.
India


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