Safety population size and duration of exposure prior to approval of new medicines: A database analysis of medicines centralised approved in the European Union between 2011 and 2023. [PDF]
Bouwman L +3 more
europepmc +1 more source
Valuing Intellectual Property Rights in an Imperfectly Competitive Market: A Biopharming Application [PDF]
Small research firms developing biotechnology applications often focus on establishing intellectual property rights (IPRs), which can then be sold to more established firms with existing market channels.
Kostandini, Genti, Mills, Bradford F.
core +1 more source
ABSTRACT The high prevalence of gastrointestinal (GI) diseases and their significant impact on the quality of life require new therapeutic strategies. The development of novel therapeutic strategies should prioritize targeting the fundamental pathophysiological mechanisms underlying these diseases, including inflammation, cellular proliferation, and ...
Sayedeh Azimeh Hosseini +7 more
wiley +1 more source
Advances in orphan drug development for alpha-1 antitrypsin deficiency: a 2025 update from the FDA and EMA. [PDF]
Höger P +8 more
europepmc +1 more source
Colliding Wars: A Systematic Review on HIV Responses in Conflict‐Affected Settings
Armed conflicts heighten HIV vulnerability among adolescent girls, refugees and displaced populations through service disruptions and health system collapse. This systematic review of 17 studies identifies promising adaptive strategies like mobile clinics and emergency stock packs to sustain HIV care.
Mona Ibrahim +12 more
wiley +1 more source
Advancing hypoparathyroidism treatment: FDA approval of Palopegteriparatide as a promising orphan drug. [PDF]
Shahid SR +4 more
europepmc +1 more source
Opportunities and challenges in protein structure prediction
Abstract Deep learning methods, particularly exemplified by AlphaFold2, have revolutionized the field of protein structure prediction—an achievement recognized by the 2024 Nobel Prize in Chemistry awarded to its core developers. Despite this remarkable achievement, the broader protein folding problem is far from solved. Key challenges—each representing
Wenkai Wang +3 more
wiley +1 more source
Informing Dose for Pediatric Rare Diseases-A Survey of Recent Orphan Drugs Approvals. [PDF]
Fletcher EP +6 more
europepmc +1 more source
Federal 340B Program Payment Scheme for Drugs Designated As Orphan Products: Congressional Clarification Needed to Close the Government-Industry Revolving Door [PDF]
Yang, Y. Tony +2 more
openaire +2 more sources
When Public Health Becomes the Weapon: Current and Prospective Consequences of the Genocide in Gaza
ABSTRACT Catastrophic humanitarian conditions during the 2023–2025 genocide in Gaza have caused a public health crisis of exceptional magnitude. This article summarizes key short‐ and long‐term health consequences for Gaza's civilian population and outlines priorities for recovery.
Therese Alexandra Evald +19 more
wiley +1 more source

