Zolgensma - Open Access .click

Anais do II Congresso Brasileiro de Biotecnologia On-line, 2022
Introdução: A atrofia muscular espinhal (AME) é uma doença autossômica recessiva, caracterizada pela fraqueza progressiva dos músculos esqueléticos e respiratórios, levando a deficiências significativas como: dificuldade para falar, andar, engolir e respirar.
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Real-world experience of gene therapy with onasemnogene-abeparvovec (Zolgensma®) for patients with SMA-type1 in UK

Journal of Neurology, Neurosurgery & Psychiatry, 2022
Retrospective review of referrals to the National-Multidisciplinary-Team (NMDT) in England (& Wales), and of the clinical records of SMAtype1 patients included for Zolgensma® therapy in the UK.Data was available for 42 patients: 13, 12, 10, 6, 1 from Evelina-London, Sheffield, Bristol, Manchester and Belfast centres respectively.Patients’ age ...
Vasantha Gowda +9 more
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Health ministers condemn Novartis lottery for Zolgensma, the world’s most expensive drug

BMJ, 2020
Novartis has held the first draw to choose four babies who will receive its one-shot treatment for the genetic disease spinal muscular atrophy, Zolgensma (onasemnogene abeparvovec), amid criticism of its lottery programme from patient groups and EU health ministers. Priced in the United States at $2.1m (£1.6m; €1.9m), the most expensive drug course of
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spinal muscular atrophy
gene therapy
onasemnogene abeparvovec

nusinersen
humans
3. good health

sma
risdiplam
genetic therapy