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Pricing Zolgensma – the world’s most expensive drug

Authors : Mark Nuijten

Subjects: price valuation reimbursement; HF5001-6182; Short Communication

Source: Journal of Market Access & Health Policy, Vol 10, Iss 1 (2022)Journal of Market Access & Health Policyarticle-version (VoR) Version of Record

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Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives

Authors : Dangouloff, T; Servais, LJP

Subjects: newborn screening; risdiplam; nusinersen

Source: Therapeutics and Clinical Risk Management

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Onasemnogene Abeparvovec (AVXS-101) for the Treatment of Spinal Muscular Atrophy

Authors : Hillary Calderon; Aimen Naveed

Subjects: 0303 health sciences; business.industry; Genetic enhancement

Source: J Pediatr Pharmacol Ther

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Spinal muscular atrophy - onasemnogene abeparvovec and other therapeutic options

Authors : Anna Warych; Mikołaj Szoszkiewicz; Aleksandra Majchrzak-Celińska

Subjects: smn2; Pharmacology; smn1

Source: Farmacja Polska, Vol 76, Iss 1, Pp 10-17 (2020)

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Gene Therapy in ALS and SMA: Advances, Challenges and Perspectives

Authors : Jan Lejman; Kinga Panuciak; Emilia Nowicka

Subjects: Inorganic Chemistry; Organic Chemistry; General Medicine

Source: International Journal of Molecular Sciences. 24:1130

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An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models

Authors : Małgorzata Biernikiewicz; Michał Pochopień; Emilie Clay

Subjects: economic evaluation; HF5001-6182; business.industry

Source: Journal of Market Access & Health Policyarticle-version (VoR) Version of RecordJournal of Market Access & Health Policy, Vol 9, Iss 1 (2021)

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Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy

Authors : Tejal Aslesh; Toshifumi Yokota

Subjects: spinal muscular atrophy (SMA); Motor Neurons; QH301-705.5

Source: Cells, Vol 11, Iss 417, p 417 (2022)

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فلترة النتائج

Pricing Zolgensma – the world’s most expensive drug

Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives

Onasemnogene Abeparvovec (AVXS-101) for the Treatment of Spinal Muscular Atrophy

Spinal muscular atrophy - onasemnogene abeparvovec and other therapeutic options

Gene Therapy in ALS and SMA: Advances, Challenges and Perspectives

An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models

Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy

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