Results 161 to 170 of about 1,989 (180)

Zolgensma: The multiple and conflicting valuation of advanced therapies for rare diseases

, 2020
International ...
Rabeharisoa, Vololona, Doganova, Liliana
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P10 Zolgensma infusion and clinical progress in pharmaceutically naive SMA1 infants

Neuromuscular Disorders, 2023
H. Lee, J. Oh
openaire   +1 more source

P233 Zolgensma in spinal muscular atrophy: a Toronto paediatric hospital experience

Neuromuscular Disorders, 2023
E. Nigro, H. Gonorazky
openaire   +1 more source

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
Eline Krijkamp, Aukje K Mantel-Teeuwisse   +1 more
exaly  

NICE recommends Zolgensma for type 1SMA in infants

PharmacoEconomics & Outcomes News, 2021
openaire   +1 more source

Novartis lottery for Zolgensma draws condemnation

PharmacoEconomics & Outcomes News, 2020
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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, Austin R. Nelson, Jane Hsi, Jon Zachary, Lindsay Potts, Paul Chipman, Mohammad Ghanem, Neeta Khandekar, Ian E. Alexander, Grant J. Logan, Juha T. Huiskonen, Robert McKenna   +11 more
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NICE recommends Zolgensma for type 1 spinal muscular atrophy

PharmacoEconomics & Outcomes News, 2021
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
Nayana Prabhu, Vikki Stefans
exaly  

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