Novartis’ AveXis unit has given its reasons for refusing to supply its spinal muscular atrophy gene therapy Zolgensma to a seriously ill child in Belgium, saying compassionate use rules do no ...

gene therapy company AveXis has released promising new safety and efficacy data. AVXS-101 is being developed to treat spinal muscular atrophy (SMA) Type 1, a severe neuromuscular disease ...

At the event, AveXis Scientific Founder and Chief Scientific Officer Brian Kaspar will discuss a revolutionary, single dose gene therapy that has been developed to treat the root cause of spinal ...

At AveXis, a Novartis company ... Our current AAV9 portfolio includes gene therapy candidates for SMA, Rett syndrome, SOD1 amyotrophic lateral sclerosis (ALS), and Friedreich’s ataxia, with ...

After an associate’s twins were born with SMA, he said he became convinced that Kaspar’s gene therapy was the answer. Carbona remade BioLife into AveXis: Av for adeno-associated virus serotype ...

The pharmaceutical company says it will continue to operate its production facility near Research Triangle Park.

Intrathecal treatment with a vector-based gene therapy ... adults with SMA receive chronic SMA therapies, there is still a critical unmet need for a single-administration therapy that helps ...

with the aim of offering OAV101 IT as a one-time treatment option to SMA patients. If regulatory filings be successful, Novartis anticipates the potential for this investigational gene therapy to ...

Novartis' OAV101 IT showed a 2.39-point HFMSE improvement in SMA patients vs ... OAV101 IT is an investigational gene replacement therapy designed to directly address the genetic root cause ...

On Wednesday, Novartis gave a more detailed look at long-awaited clinical data that the company believes will help secure a broad approval for a successor drug to its blockbuster gene therapy ...