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Treatment with salanersen slowed neurodegeneration and improved motor function in children with SMA in a Phase 1 trial, ...
BiogenBIIB said Wednesday its experimental treatment helped children with a devastating muscle-wasting disease even after they received an approved gene therapy from NovartisNVS.The company already ...
The Columbus startup received FDA approval and has a clinical trial underway for a therapy targeting acute lymphoblastic ...
The therapy has a high response rate, he said. The cost of a traditional ongoing treatment for spinal muscular atrophy, or “floppy baby syndrome” is about $300,000 to $400,000 a year, Lennon said.
Once Novartis acquired AveXis, it turned to setting a price for its much-anticipated gene therapy. Unlike other nations, the United States allows companies to charge whatever they want for new drugs.
Novartis is set to launch its one-off gene therapy Zolgensma for the ultra-rare muscle-wasting disease spinal muscular atrophy (SMA) in Germany at the start of July priced at 1,945,000 euro R&D ...
AveXis purchased rights to its gene therapy technology in 2015 for an undisclosed sum from Asklepios BioPharmaceutical, a Chapel Hill gene therapy platform company commonly called AskBio.
AveXis, a Novartis company, announced the European Commission (EC) granted conditional approval for Zolgensma (onasemnogene abeparvovec) for the treatment of patients with 5q spinal muscular atrophy ...
Novartis’ gene therapy arm AveXis has announced European approval for Zolgensma (onasemnogene abeparvovec), the only gene therapy for spinal muscular atrophy (SMA). Specifically, the European ...
Cite this: Megan Brooks. EMA Panel Backs Zolgensma Gene Therapy for SMA - Medscape - Mar 27, 2020.
Interim Phase I/II data for Zolgensma showed improvements in children aged 2 to less than 5 with spinal muscular atrophy. The therapy received FDA approval in May for infants younger than 2.