Results 81 to 90 of about 29,818 (295)
Kenya’s Life Lessons through the Lived Experience of Rural Caregivers
This qualitative research study used a phenomenological lens to examine the perspectives of familial caregivers in the Laikipia Region of Kenya. Through the narrative of the caregivers’ lived experience, key factors identified included social supports ...
Amy Cappiccie +2 more
doaj +1 more source
Introduction. Drugs used for treatment of rare diseases are known worldwide under the term of orphan drugs because pharmaceutical companies have not been interested in ?adopting? them, that is in investing in research, developing and producing these drugs.
Svetlana Golocorbin-Kon +4 more
openaire +3 more sources
Regulators use expedited approval pathways to speed market approval and patient access to promising new drugs. However, there is uncertainty about whether these pathways are successful in approving drugs with significant therapeutic advantages. This systematic review aims to examine the safety, effectiveness and cost‐effectiveness of drugs approved via
Ashleigh Hooimeyer +4 more
wiley +1 more source
You’re an Orphan When Science Fiction Raises You
In Among Others, Jo Walton’s fairy story about a science-fiction fan, science fiction as a genre and archive serves as an adoptive parent for Morwenna Markova as much as the extended family who provide the more conventional parenting in the absence of ...
Halpin Jenni G.
doaj +1 more source
Decoding RNA regulation: Challenges and opportunities for RNA‐based therapies in Europe
Abstract RNA‐based medicinal products represent a promising frontier in personalised medicine, offering sequence‐specific disease targeting at various molecular levels, yet their clinical translation in the European Union (EU) may be hindered by regulatory uncertainty around definitions and evidence requirements; this study therefore aims to identify ...
Olivia C. Lewis +4 more
wiley +1 more source
Regulatory harmonization: Evolution, globalization and future directions
Regulatory harmonization has become an increasingly important and accepted approach to streamline regulatory review processes and expedite access to safe, effective and high‐quality medicines globally. This review explores the evolution and current status of regulatory harmonization, convergence and reliance initiatives.
Orin Chisholm +2 more
wiley +1 more source
Anxiety, Depression, Stress, and Decision-Making Among Orphans and Non-Orphans in Pakistan
Farah Shafiq,1 Sonia Ijaz Haider,2 Shamaila Ijaz1 1Lahore Institute of Special Care and Attention (LISCA-Private Clinic), Lahore, Punjab, Pakistan; 2Department for Educational Development, The Aga Khan University, Karachi, Sindh, PakistanCorrespondence ...
Shafiq F, Haider SI, Ijaz S
doaj
Lifecycle perspective on cell and gene therapy manufacturing challenges and enabling GMP solutions. ABSTRACT Cell and gene therapies (CGTs) are revolutionizing modern medicine; however, making these advanced medicines scalable and readily available to commercial manufacturers worldwide is a major challenge. The number of approved CGT products continues
Rajath Samaga +2 more
wiley +1 more source
Adults with LCH – orphans with an orphan disease [PDF]
Tatevossian R +6 more
openaire +2 more sources

