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Synthesis and use of polyamine derivatives as internal mass spectrometry standards [PDF]
Dar, Sami Ullah
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siRNA Therapeutics for the Treatment of Hereditary Diseases and Other Conditions: A Review. [PDF]
Int J Mol SciShevelev A +3 more
europepmc +1 more source
Mode 2 knowledge production: evidence from orphan drug networks
Science and Public Policy, 2007 This paper seeks to contribute to the understanding of public participation in the process of science policy-making and the lay public's contribution to scientific knowledge production. Nowotny et al (2001) claim that the traditional boundaries between expert and lay knowledge are being transgressed and science and technology are thereby enriched.
Helen Crompton
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Europe finally agrees to encourage orphan-drug production
The Lancet, 2000 Arthur Rogers
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Plant molecular farming: a promising frontier for orphan drug production
Biotechnology LettersOrphan diseases comprise a range of disorders that individually affect a small percentage of people, but collectively impact millions of people worldwide. Patients with this disorder often face significant challenges in diagnosis, treatment, and access to care due to their rare nature and limited understanding and treatment options.
Balamurugan Shanmugaraj +2 more
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International Journal of Technology Assessment in Health Care, 2022
IntroductionIn 2018 the Dutch Ministry of Health (MoH) introduced a new policy regarding the conditional reimbursement (CR) of drugs in the basic health insurance package. This new policy offers patients with a serious, often rare disease for which no effective treatment is yet available, the possibility of obtaining controlled access to new promising ...
Floor van Heesch, Marijke De Vries
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IntroductionIn 2018 the Dutch Ministry of Health (MoH) introduced a new policy regarding the conditional reimbursement (CR) of drugs in the basic health insurance package. This new policy offers patients with a serious, often rare disease for which no effective treatment is yet available, the possibility of obtaining controlled access to new promising ...
Floor van Heesch, Marijke De Vries
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Expert Opinion on Orphan Drugs, 2021
The Orphan Drug Act was created to stimulate the development of drugs and biologics for rare diseases. Investigating products that have received orphan drug designation provide a greater understanding of rare disease drug development, as well as the repositioning business models of developers.We used a dataset containing all orphan drug designations ...
Kathleen L, Miller +3 more
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The Orphan Drug Act was created to stimulate the development of drugs and biologics for rare diseases. Investigating products that have received orphan drug designation provide a greater understanding of rare disease drug development, as well as the repositioning business models of developers.We used a dataset containing all orphan drug designations ...
Kathleen L, Miller +3 more
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Orphan drug product regulation – United States
Int. Journal of Clinical Pharmacology and Therapeutics, 2002The legislative history of the United States Orphan Drug Act began with rare, unanimous approval by the United States Congress. The Act, mid consequently the Regulations, have evolved since then. The two-stage process of Orphan Drug designation and approval is outlined, as well as the incentives that are offered to commercial companies for their ...
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