Results 21 to 30 of about 3,694,565 (212)
British Journal of Clinical Pharmacology, EarlyView.Introduction In recent years, the treatment of spinal muscular atrophy (SMA), a rare disease, has significantly progressed, improving patients' survival and overall quality of life. However, current SMA treatments are expensive, and some (nusinersen) are very inconvenient for patients.
Andrej Belančić +4 more
wiley +1 more source
Developing Methodology for the Creation of Clinical Practice Guidelines for Rare Diseases : A Report from RARE-Bestpractices [PDF]
, 2015 Date of Acceptance: 29/05/2015 The research leading to these results has received funding from the (FP7/2007–2013), under grant agreement n 305690 (RARE-Bestpractices project). The opinions, presented here reflect only the authors’ views.
RARE-Bestpractices Consortium
core +1 more source
British Journal of Clinical Pharmacology, EarlyView.Aims Regulatory inconsistency across Africa causes duplicative assessments, prolonged approvals, and delayed medical access. To inform the African Medicines Agency (AMA), the African Medicines Regulatory Harmonisation (AMRH) piloted Africa's first continental scientific evaluation of human medicinal products.
Alex Juma Ismail +4 more
wiley +1 more source
RDA & Rare Materials Seminar, Edinburgh, 6th November
, 2015 CIG Scotland is delighted to host an international seminar on RDA & Rare Materials, in conjunction with members of the Association of College & Research Libraries’ Rare Books and Manuscripts Section (RBMS), the European RDA Interest Group (EURIG), the ...
IFLA Rare Books & Special Collections Section
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British Educational Research Journal, EarlyView.Abstract All children should have access to quality education through a child‐centred pedagogy. An inclusive, child‐centred pedagogy uses a strength‐based view of children that recognises each child as unique and competent, providing children with multiple opportunities to explore and learn at their own pace.
Katherine Gulliver
wiley +1 more source
Moderators' perceptions of consistency in Key Stage 2 writing moderation across local authorities
British Educational Research Journal, EarlyView.Abstract This article explores moderators’ perceptions of the consistency of Key Stage 2 (KS2) writing moderation across Local Authorities (LAs) in England, a process central to securing the reliability and fairness of teacher assessment in a high‐stakes accountability system.
Rebecca Clarkson
wiley +1 more source
British Educational Research Journal, EarlyView.Abstract This article presents the first systematic review of scholarship applying Pierre Bourdieu's theoretical framework in New Zealand educational research, drawing on 30 peer‐reviewed studies published between 2000 and 2025. Following PRISMA guidelines, studies were coded for Bourdieusian concepts applied, depth of theoretical application ...
Liuning Yang
wiley +1 more source
Registration OPEN: “A common international standard for rare materials cataloguing? Why? And how?”
, 2016 “A common international standard for rare materials cataloguing? Why? And how?” 22nd February 2016, Biblioteca Nacional de Portugal (Lisbon), 10am-6pm Registration is now open for this one-day conference being held at the Biblioteca Nacional de Portugal (
IFLA Rare Books & Special Collections Section
core +1 more source
Food‐solicitation displays in nestling birds: still begging for answers
Biological Reviews, EarlyView.ABSTRACT Nestling begging is a key model for understanding animal communication and parent–offspring conflict. Dynamic begging displays are often interpreted as costly, honest signals of need, with honesty maintained by viability costs, as proposed in Godfray's influential signalling model.
Daniel Parejo‐Pulido, Tomás Redondo
wiley +1 more source
Business Strategy and the Environment, EarlyView.ABSTRACT The relationship between eco‐innovation (EI) and firm performance (FP) is central to environmental management research, yet empirical evidence remains fragmented and highly context dependent. This article presents a global systematic literature review (SLR) of 541 empirical studies (2006–April 2025) to (i) diagnose the systematic sources of ...
Ali Mammadov
wiley +1 more source