Results 141 to 150 of about 2,159 (173)
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Amyotrophie spinale : l’affaire du zolgensma de novartis (1)
Revue Medicale Suisse, 2020exaly +2 more sources
The Zolgensma Journey: A Groundbreaking Therapy for SMA
Iulian Alecu, Nayla Mumnehexaly +2 more sources
ZOLGENSMA: TERAPIA GÊNICA PARA A ATROFIA MUSCULAR ESPINHAL
Anais do II Congresso Brasileiro de Biotecnologia On-line, 2022Introdução: 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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Health ministers condemn Novartis lottery for Zolgensma, the world’s most expensive drug
BMJ, 2020Novartis 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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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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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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Real-World Data for Onasemnogen Abeparvovec (Zolgensma) in Spinal Muscular Atrophy
Neuropediatrics, 2021C. Weiß +29 more
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P233 Zolgensma in spinal muscular atrophy: a Toronto paediatric hospital experience
Neuromuscular Disorders, 2023E. Nigro, H. Gonorazky
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International Journal of Drug Delivery Technology
Spinal Muscular Atrophy (SMA) is a rare, progressive, autosomal recessive neuromuscular disorder characterized by degeneration of alpha motor neurons in the spinal cord, resulting in muscle weakness, respiratory compromise, and severe disability. The condition is primarily caused by mutations or deletions in the SMN1 gene, leading to insufficient ...
Shaik Asha Begum +6 more
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Spinal Muscular Atrophy (SMA) is a rare, progressive, autosomal recessive neuromuscular disorder characterized by degeneration of alpha motor neurons in the spinal cord, resulting in muscle weakness, respiratory compromise, and severe disability. The condition is primarily caused by mutations or deletions in the SMN1 gene, leading to insufficient ...
Shaik Asha Begum +6 more
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P10 Zolgensma infusion and clinical progress in pharmaceutically naive SMA1 infants
Neuromuscular Disorders, 2023H. Lee, J. Oh
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