Results 71 to 80 of about 124,112 (267)
Decoding RNA regulation: Challenges and opportunities for RNA‐based therapies in Europe
British Journal of Clinical Pharmacology, EarlyView.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
Hematology Reports, 2015 Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-orphan disease affecting all hematopoietic cell types. The abnormality of red blood cells in this disease predisposes to intravascular complement-mediated hemolysis.
Hassan A. Al-Jafar +4 more
doaj +1 more source
, 1977 Series VIII: Audiocassettes features informal oral histories about the Hebrew Orphan Asylum and its alumni.Digital recordingDigital finding ...
Hebrew Orphan Asylum of New York
core
Regulatory harmonization: Evolution, globalization and future directions
British Journal of Clinical Pharmacology, EarlyView.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
Pulmonary TherapyIntroduction Cystic fibrosis (CF) transmembrane conductance regulator modulators (CFTRm) have transformed CF care, shifting treatment from only managing symptoms to also addressing the underlying defects that cause CF.
Jaime L. Rubin +5 more
doaj +1 more source
HematologyObjectives Our aim was to better understand and raise awareness of the diagnosis journey and quantify any barriers for timely diagnosis of haemophagocytic lymphohistiocytosis (HLH), to support patients’ struggle with diagnosis and reduce time to ...
Tracy Machado +3 more
doaj +1 more source
Frontiers in Psychiatry, 2019 The negative effects of institutionalization on children’s wellbeing and psychological adjustment have been extensively documented. Throughout the world, particularly in developing countries, many children in residential child care institutions known as ...
Epaphrodite Nsabimana +4 more
doaj +1 more source
Baron, Shirley in her home (Box 90, Folder 6)
, 1988 Series VIII: Audiocassettes features informal oral histories about the Hebrew Orphan Asylum and its alumni.Digital recordingDigital finding ...
Hebrew Orphan Asylum of New York
core
Biotechnology and Bioengineering, EarlyView.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
Mechanism of Penoxsulam’s Effect on Chlorophyll Synthesis and the Metabolism of Foxtail Millet
PlantsFoxtail millet is a characteristic miscellaneous grain crop with many benefits in current agricultural production and is crucial in the adjustment of the planting structure and the sustainable development of dry farming.
Tingting Chen +9 more
doaj +1 more source