HAVING A GO AT SPINAL MUSCULAR ATROPHY WITH SPINRAZA
Keywords:Spinraza, Spinal muscualr atrophy, Antisense oligonucleotide, Adverse effects, Endear trial
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.
Faravelli I, Nizzardo M, Comi GP, Corti S. Spinal muscular atrophy â€“ Recent therapeutic advances for an old challenge. Nat Rev Neurol 2015;11(6):351-9.
U.S. FDA Approves Biogenâ€™s SPINRAZAâ„¢ (Nusinersen), the First Treatment for Spinal Muscular Atrophy [News release]. Boston, MA: Biogen; December 23; 2016. Available from: http://www.media. biogen.com/press-release/neurodegenerative
-diseases/us-fda-approves-biogensspinraza- nusinersen-first-treatment. [Last accessed on 2016 Dec 23].
Biogen. SPINRAZA. Prescribing Information. Cambridge, MA: Biogen; December; 2016.
Chiriboga CA, Swoboda KJ, Darras BT, Iannaccone ST, Montes J, De Vivo DC, et al. Results from a phase 1 study o f nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy. Neurology 2016;86(10):890-7.
HachÃ© M, Swoboda KJ, Sethna N, Farrow-Gillespie A, Khandji A, Xia S, et al. Intrathecal injections in children with spinal muscular atrophy: Nusinersen clinical trial experience. J Child Neurol 2016;31(7):899-906.
Kuntz N, Farwell W, Zhong ZJ, Sun P, Gheuens S, Schneider E, et al. Nusinersen treatment of infantile-onset spinal muscular atrophy (SMA): Study design and initial interim efficacy and safety findings from the phase 3 ENDEAR study. World Muscle Society Meeting, Granada Spain, October; 2016.
Glanzman AM, Mazzone E, Main M, Pelliccioni M, Wood J, Swoboda KJ, et al. The Childrenâ€™s Hospital of Philadelphia Infant test of neuromuscular disorders (CHOP INTEND): Test development and reliability. Neuromuscul Disord 2010;20(3):155-61.
How to Cite
The publication is licensed under CC By and is open access. Copyright is with author and allowed to retain publishing rights without restrictions.