The temporalities of prices: 'Value-based pricing' in pharmaceutical markets
International audienceIn 2019; Zolgensma, a gene therapy for patients suffering from Spinal Muscular Atrophy (SMA), became famous for being 'the most expensive drug ever.' Its high price was justified by 'the value' that the drug will bring to patients ...
Doganova, Liliana, Rabeharisoa, Vololona
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Securitization as a means to pay for cell and gene therapies for orphan diseases: a simulation study. [PDF]
Gene TherLu JM +3 more
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External controls for rare disease drug development: Lessons for emerging and advanced therapeutic modalities. [PDF]
Mol Ther AdvHughes SH +6 more
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Small populations, big impact: leveraging rare disease gene therapies to benefit millions. [PDF]
J Clin InvestCherqui S.
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Broadening applications for intrathecal gene therapy: a case for lysosomal storage diseases. [PDF]
EBioMedicineeBioMedicine.
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New hope for older SMA patients with next-generation self-complementary AAV gene therapy. [PDF]
Mol TherNakai H, Finanger EL, Samulski RJ.
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Türkiye’de SMA’lı Çocukların Tedavisi ve Yabancı Ülke Örnekleri
SMA hastalığı nadir görülen bir gen hastalığıdır. SMA için şu anda uygulanmakta olan bir ilaç tedavisi mevcuttur. Son dönemlerde yapılan bu ilaç tedavisine “Zolgensma” isimli yeni ve pahalı bir ilaç daha eklenmiştir.
Arslan Ertürk, Arzu
core
A Cost-Effectiveness Analysis for Treatments of Patients with Early Stage Huntington's Disease in the USA. [PDF]
Pharmacoecon OpenPatil D +3 more
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Commentary: Current Status of Gene Therapy for Spinal Muscular Atrophy
Frontiers in Cellular Neuroscience, 2022 Wilfried Rossoll, Ravindra N. Singh
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How much do ex-US revenues make a difference for pharmaceutical investment returns? [PDF]
J Comp Eff ResRamagopalan SV +3 more
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