Zolgensma - Open Access .click

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NICE recommends Zolgensma for type 1SMA in infants

PharmacoEconomics & Outcomes News, 2021
openaire +1 more source

Structural characterization of antibody-responses from Zolgensma treatment provides the blueprint for the engineering of an AAV capsid suitable for redosing

Abstract Monoclonal antibodies (mAbs) are useful tools to dissect the neutralizing antibody response against the adeno-associated virus (AAV) capsids used as gene therapy delivery vectors. This study structurally characterizes the interactions of 21 human-derived antibodies from patients treated with the AAV9 vector ...
Mario Mietzsch +11 more
openaire +2 more sources

Towards sustainability and affordability of expensive cell and gene therapies? Applying a cost-based pricing model to estimate prices for Libmeldy and Zolgensma

Cytotherapy, 2022
Carin Uyl-De Groot +2 more
exaly

Early Cost-Effectiveness of Onasemnogene Abeparvovec-xioi (Zolgensma) and Nusinersen (Spinraza) Treatment for Spinal Muscular Atrophy I in The Netherlands With Relapse Scenarios

Value in Health, 2021
Rick Vreman, Wim Goettsch
exaly

Novartis lottery for Zolgensma draws condemnation

PharmacoEconomics & Outcomes News, 2020
openaire +1 more source

NICE recommends Zolgensma for type 1 spinal muscular atrophy

PharmacoEconomics & Outcomes News, 2021
openaire +1 more source

HPR55 Reimbursement of High Priced One-Shot Treatments in Korea: KYMRIAH and ZOLGENSMA Case Study

Value in Health, 2023
E. Shin, S. Kim, J. Koo
openaire +1 more source

Thrombotic Microangiopathy (TMA): A potential adverse reaction post Zolgensma (onasemnogene abeparvovec-xioi) therapy for Spinal Muscular Atrophy (SMA) (5483)

Neurology, 2020
Amit Agarwal +2 more
exaly