Epidemiological characteristics and natural history of porphyria - a twenty-year population-based analysis in Taiwan. [PDF]
Orphanet J Rare DisWu WC +10 more
europepmc +1 more source
Methodological challenges in Dutch HTA of non-oncological orphan drugs: a retrospective analysis and price comparison using different pricing models. [PDF]
Orphanet J Rare DisWalraven J, Kaveh M, Uyl-de Groot C.
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04103 Effect of Givosiran for hereditary coproporphyria: 2 cases report
Poster 9Tomohide Adachi, Nobuaki Ozaki
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Neuralgic amyotrophy presentation of acute intermittent porphyria: A case report. [PDF]
J Peripher Nerv SystTheuriet J +4 more
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Confidential and non-confidential drug rebates in Switzerland, 2013–2023: empirical analysis of drug rebates, trends over time and re-evaluations [PDF]
Herrle, Cascal +3 more
core +1 more source
Fair Funding Decisions: Consistency of the Time Horizons Used in the Calculation of Quality-Adjusted Life Years for Therapies for Very Rare Diseases by the National Institute for Health and Care Excellence in England. [PDF]
Int J Environ Res Public HealthBarman-Aksözen J +7 more
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Advancing hypertension management: the role of zilebesiran as an siRNA therapeutic agent. [PDF]
Ann Med Surg (Lond)Siddiqui E +5 more
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04148 Single-center real-world experience with givosiran for acute intermittent porphyria
Session 6 – Current therapies for acute hepatic porphyrias (AHP) (September 24th, 2024)Chanan Stauffer +3 more
openaire +1 more source
Comment on: "An Early Cost-Utility Model of mRNA-Based Therapies for the Treatment of Methylmalonic and Propionic Acidemia in the United Kingdom". [PDF]
Clin Drug InvestigPerera S +8 more
europepmc +1 more source