Ann Neurol. Le gel d'un programme clinique n'en constitue pas moins en soi une mauvaise nouvelle.

Novartis a acquis l'an dernier Avexis, qui développe le Zolgensma, pour près de neuf milliards de francs suisses. 2018; This website uses cookies to personalize your content (including ads), and allows us to analyze our traffic. The retrospectively-estimated dosage range in the high-dose cohort is approximately 1.1 × 10[1][4] to 1.4 × 10[1][4] vg/kg. Younger SMA patients need the muscle-boosting protein Zolgensma helps create to be expressed throughout the body, while older patients require a more targeted approach, which is why Novartis is developing the intrathecal version for them. IndicationZolgensma (onasemnogene abeparvovec-xioi) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patient less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. OneGene ProgramTM, AveXis' comprehensive patient support program, provides a dedicated, personalized support team focused on the needs of each family throughout the Zolgensma treatment journey.

Novartis avait dans la foulée fait le ménage dans les équipes de recherche d'Avexis, remerciant les responsables incriminés. In the interim, AveXis has arranged to make the product available for international markets, subject to local laws and regulations, as a part of its paid Managed Access Program via a collaboration with Durbin, a third-party provider. About Zolgensma Clinical DataThe efficacy of Zolgensma in pediatric patients less than 2 years of age with SMA with bi-allelic mutations in the SMN1 gene was evaluated in STR1VE, an open-label, single-arm clinical trial (ongoing), and in START, an open-label, single-arm, ascending-dose clinical trial (completed). 365 Bloor Street East, Toronto, Ontario, M4W 3L4. Novartis, which spent $8.7 billion for U.S.-based AveXis in 2018 to get Zolgensma, which is the world’s most-expensive one-time treatment, said it remained confident in the gene therapy’s benefit-risk profile. Unauthorized distribution, transmission or republication strictly prohibited. There was an error, please provide a valid email address.

Before treatment with Zolgensma, none of the 21 patients required non-invasive ventilator (NIV) support, and all patients could exclusively feed orally (i.e., no need for non-oral nutrition). 2017 Jul;264(7):1465-1473. Nosotros y nuestros socios almacenaremos y/o accederemos a la información de tu dispositivo mediante el uso de cookies y tecnologías similares, a fin de mostrar anuncios y contenido personalizados, evaluar anuncios y contenido, obtener datos sobre la audiencia y desarrollar el producto. Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time intravenous (IV) infusion.