BioPharma Dive (WEB 01/16/2020)
SAN FRANCISCO — Novartis sparked a new round of drug pricing criticism last May when the Swiss pharma revealed its new gene therapy Zolgensma would cost $2.1 million per patient.
Pipeline products to prioritize: "Those programs existed as part of the acquisition of AveXis. Since then, we started partnering with [the Novartis Institutes of Biomedical Research] to apply this platform to different diseases. The first one we announced is a project for Friedreich's ataxia, a muscle-wasting disease."
Thursday, January 16, 2020
Safety and Efficacy of Interferon γ in Friedreich's Ataxia
Marinela Vavla MD PhD Maria Grazia D'Angelo MD PhD Filippo Arrigoni MD Nicola Toschi PhD Denis Peruzzo PhD Sandra Gandossini MD Annamaria Russo MD Eleonora Diella Stefania Tirelli PhD Roberto Salati MD Paolo Scarpazza MD Riccardo Luffarelli PhD Silvia Fortuni PhD Alessandra Rufini PhD Ivano Condò PhD Roberto Testi MD Andrea Martinuzzi MD PhD; Mov Disord. 2020 doi:10.1002/mds.27979
A relevant indication that emerges from the study, the previously studies, and from the numerous FRDA patients using IFNγ off label is the possible presence of nonresponders. A randomized withdrawal design is therefore best suited to increase the power of future clinical trials to definitively assess the efficacy of IFNγ treatment in FRDA
A relevant indication that emerges from the study, the previously studies, and from the numerous FRDA patients using IFNγ off label is the possible presence of nonresponders. A randomized withdrawal design is therefore best suited to increase the power of future clinical trials to definitively assess the efficacy of IFNγ treatment in FRDA
Subscribe to:
Posts (Atom)
