Pharmaceuticals, Biotechnology and Life Sciences
Novartis’ company Avexis said that it’s Phase 1/2 Srong study for intrathecal (IT) administration of AVXS-101, showed that patients aged 2 to 5, with spinal muscular atrophy (SMA) Type 2 achieved a mean increase of 5.9 points from baseline in HFMSE scores, nearly double the clinically meaningful threshold, at a mean duration of follow-up time of 9.3 months.
Novartis’s Biologics License Application (BLA) for Zolgensma, designed to tackle the genetic root cause of spinal muscular atrophy (SMA) Type 1, a deadly neuromuscular disease, was accepted by the U.S. Food and Drug Administration (FDA).
Genomic Vision, DNA molecular combing specialist that develops tests for the diagnostics market and tools for the life sciences research market, has presented, at the Annual Meeting of the American Society of Human Genetics, the initial results of the pilot study undertaken with Quest Diagnostics that aims to identify a biomarker to improve genetic counseling for Spinal Muscular Atrophy (SMA) in the African-American population.