Sustained Effectiveness of Lanadelumab in Preventing Hereditary Angioedema Attacks: The ENABLE Study
ABSTRACT Background Lanadelumab has been approved for hereditary angioedema (HAE) long‐term prophylaxis since 2018. The Phase 4, prospective ENABLE Study (NCT04130191) evaluated the long‐term effectiveness and safety of lanadelumab in clinical practice across Europe and the Middle East.
Andrea Zanichelli +15 more
wiley +1 more source
GPR3 Ligands Discovered through Combined Virtual and Conformational Biosensor-Based Screening. [PDF]
Schihada H +14 more
europepmc +1 more source
A Systematic Review on Disease‐Modifying Therapies in Parkinsonian Disorders
Parkinsonian disorders, including Parkinson's disease, Lewy body dementia, multiple system atrophy, and progressive supranuclear palsy, are progressive neurodegenerative conditions with no treatment options to slow disease progression. This systematic review provides an overview of evidence of disease‐modifying therapies that have been evaluated in ...
Pepijn P.N.M. Eijsvogel +3 more
wiley +1 more source
Can the Development of Orphan Drugs Include Wider Patient Engagement? A Citizens' Jury to Explore a Promissory Notion. [PDF]
Frost J +5 more
europepmc +1 more source
A Tale of Two Mechanisms: The p53 Modulator COTI‐2 Is a Zn Metallochaperone
ABSTRACT Mutations in, or misregulation of, Tp53 are found in approximately 50% of all cancers. p53 functions by ensuring that cells with irretrievably damaged DNA undergo apoptosis. Tp53 mutations often induce conformational changes that inhibit activity; however, small‐molecule chaperones could theoretically restore conformation and activity. COTI‐2,
İrem Şimşek +16 more
wiley +1 more source
Consequences of Canada's Drug Agency Reimbursement Recommendations for New Medicines and Pan-Canadian Pharmaceutical Alliance Price Negotiations on Patient Access. [PDF]
Rawson NSB.
europepmc +1 more source
2′‐FL's Depiction of the Human Physiological Function Landscape
The figure summarizes the multifaceted physiological effects of 2′‐fucosyllactose (2′‐FL), a major human milk oligosaccharide. The schematic illustrates the central role of the gut‐centered regulatory axis, where 2′‐FL modulates gut microbiota composition, promotes the production of short‐chain fatty acids and microbial metabolites, enhances mucus ...
Yushan Xiao +3 more
wiley +1 more source
Early and accelerated access programs for medical devices in the European Union: mapping regulatory derogations and national schemes. [PDF]
Haon B +4 more
europepmc +1 more source
ABSTRACT Background and Aims Atherosclerosis is a lipid‐induced chronic inflammatory cardiovascular disorder denoted by the infiltration of immune cells and the agglomeration of lipids in the intima, the innermost layer, of the arteries of large and medium sizes.
Ramin Lotfi +4 more
wiley +1 more source
Pathing the way from regulatory approval to market access for gene therapy products (GTPs): An integrative review in the US, EU5, Japan and China. [PDF]
Shi J, Hu H, Ung COL.
europepmc +1 more source

