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AveXis presents data from ongoing Zolgensma trials in SMA patients

Author : Paroma Bhattacharya | Published Date : 2019-05-06 
  • The incidence of SMA is one in every 10,000 live births.
  • SMA is often recognized as the leading genetic cause behind infant mortality.

AveXis, the Novartis-held biotechnology company based in the U.S., has reportedly declared data from the ongoing trials of Zolgensma (onasemnogene abeparvovec-xioi, AVXS-101) that exhibited positive results in a broad selection of patients afflicted with SMA (Spinal Muscular Atrophy).

Authentic sources close to the matter stated that the data includes the first presentation from the Phase 1 STRONG trial that exhibited motor function gains & milestone achievements through IT (intrathecal) delivery in patients with SMA Type 2; new data from Phase 3 STR1VE trial, which showed increase in motor functions and prolonged event-free survival, and first data presentation from the Phase 3 SPR1NT trial, that showed motor milestone achievement coupled with normal development in SMA patients who are treated pre-symptomatically. Sources further added that these data have been presented at the AAN (American Academy of Neurology) 2019 Annual Meeting.

SMA, for those uninitiated, is a critical neuromuscular disorder characterized by the loss of motor neurons resulting in progressive muscle weakness & paralysis. It generally occurs due to a defect in the SMN1 gene which codes SMN – a significant protein for survival of motor neurons.

According to David Lennon, President, AveXis, the company has witnessed Zolgensma offering speedy motor function improvement, prolonged survival and milestone achievements with a single, one-time dose. He went on to add that the robust data presented at the AAN signifies a growing body of evidence supporting Zolgensma’s usage as a potential, foundational therapy for the SMA treatment across a huge population.

Zolgensma is an investigational gene therapy which is in development at present as a one-time infusion for the SMA Type1. It was developed in close collaboration with Genethon, and in December 2018, the Food & Drug Administration (FDA) approved the Biologics License Application of the company for use of Zolgensma in in SMA Type 1 patients.

Source Credits: https://www.prnewswire.com/news-releases/avexis-presented-robust-data-at-aan-demonstrating-efficacy-of-zolgensma-in-broad-spectrum-of-spinal-muscular-atrophy-sma-patients-300843943.html


About Author

Paroma Bhattacharya

Paroma Bhattacharya

Paroma Bhattacharya, having completed her post-graduation in Journalism and Mass Communication, started her career in writing with resourceful and informative content development across diverse fields. Having dealt in myriad topics ranging from business to real estate, she now pens down articles for fractovia.org and other portals. She can be contacted at- [email protected] | https://twitter.com/paromab1

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