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A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma (ARISER)

• Source: A Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) - ARISER

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ID236 Zolgensma no tratamento da Atrofia Muscular Espinhal: Acordos de Acesso Gerenciado no mundo

• Authors : Laís Lessa Neiva Pantuzza; Amanda Oliveira Lyrio; Ana Carolina de Freitas Lopes

Subjects: Acordo de Acesso Gerenciado; Zolgensma; Onasemnogeno abeparvoveque

• Source: Jornal de Assistência Farmacêutica e Farmacoeconomia, Vol 9, Iss s. 1 (2024)

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Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma ®) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System.

• Authors : Ruggiero, Rosanna^1,2 (AUTHOR) ; Balzano, Nunzia^1,2 (AUTHOR) ; Nicoletti, Maria Maddalena³ (AUTHOR)

• Source: Pharmaceuticals (14248247). Mar2024, Vol. 17 Issue 3, p394. 13p.

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Judicialization of Zolgensma in the Ministry of Health: costs and clinical profile of patients

• Authors : Ana Katheryne Miranda Kretzschmar; Ellen Teixeira; Dayani Galato

Subjects: Spinal Muscular Atrophy; Onasemnogene Abeparvovec; Health Judicialization

• Source: Revista de Saúde Pública, Vol 58 (2024)

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Experience of using gene replacement therapy with Zolgensma® (onasemnogene abeparvovec) in real clinical practice in Russia

• Authors : S. B. Artemyeva; Yu. O. Papina; O. A. Shidlovskaya

Subjects: spinal muscular atrophy; onasemnogene abeparvovec; zolgensma®

• Source: Нервно-мышечные болезни, Vol 12, Iss 1, Pp 29-38 (2022)

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Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma®) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System

• Authors : Rosanna Ruggiero; Nunzia Balzano; Maria Maddalena Nicoletti

Subjects: gene therapy; orphan drug; safety data

• Source: Pharmaceuticals, Vol 17, Iss 3, p 394 (2024)

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Pricing Zolgensma – the world's most expensive drug.

• Authors : Nuijten, Mark (AUTHOR)

Subjects: PRICES; BUSINESS negotiation; SPINAL muscular atrophy

• Source: Journal of Market Access & Health Policy. Dec2022, Vol. 10 Issue 1, p1-3. 3p.

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An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER).

• Authors : Dean, Rebecca (AUTHOR); Jensen, Ivar (AUTHOR); Cyr, Phil (AUTHOR)

Subjects: SPINAL muscular atrophy; QUALITY-adjusted life years; COST effectiveness

• Source: Journal of Market Access & Health Policy. Dec 2021, Vol. 9 Issue 1, p1-12. 12p.

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

• Authors : Thielen, Frederick W.; Heine, Renaud J.S.D.; Berg, Sibren van den

Subjects: dk/atira/pure/sustainabledevelopmentgoals/good_health_and_well_being; article; article

• Source: Thielen , F W , Heine , R J S D , Berg , S V D , Ham , R M T T & Groot , C A U D 2022 , ' Towards sustainability and affordability of expensive cell and gene therapies? Applying a cost-based pricing

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Judicialization of Zolgensma in the Ministry of Health: costs and clinical profile of patients ; Judicialização do Zolgensma no Ministério da Saúde: custos e perfil clínico dos pacientes

• Authors : Silva, Everton Nunes da; Kretzschmar, Ana Katheryne Miranda; Teixeira, Ellen

Subjects: Spinal Muscular Atrophy; Onasemnogene Abeparvovec; Health Judicialization

• Source: Revista de Saúde Pública; v. 58 n. 1 (2024); 36 ; Revista de Saúde Pública; Vol. 58 Núm. 1 (2024); 36 ; Revista de Saúde Pública; Vol. 58 No. 1 (2024); 36 ; 1518-8787 ; 0034-8910

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