Results 231 to 240 of about 124,112 (267)

Use of Real-World Evidence in Health Technology Assessments of Non-Oncology Rare Disease Therapies. [PDF]

J Mark Access Health Policy
Blandy O, Lembo P, Folorunso R, Myren KJ, Chevrou-Severac H, Thomas SK.   +5 more
europepmc   +1 more source

Reimagining the White Child as Other: Taika Waititi's Decolonial Play in James and the Giant Peach With Taika and Friends

Children &Society, EarlyView.
ABSTRACT This article examines how James and the Giant Peach with Taika and Friends (JGPTF) (2020) challenges the colonial logics embedded in Roald Dahl's construction of the universal white child. Through collaborative digital play, humour and Māori relational epistemologies, Taika Waititi unsettles James's position as the normative child of futurity,
Diana Sandars
wiley   +1 more source

Drug and country characteristics associated with global approval and launch of FDA-approved novel therapeutics. [PDF]

Health Aff Sch
Fu M, Wallach JD, So AD, Doernberg H, Li X, Guan X, Ramachandran R, Ross JS.   +7 more
europepmc   +1 more source

Expanding Patient Access to Advanced Therapies. [PDF]

BioDrugs
Guillen E, Hidalgo-Simon A.
europepmc   +1 more source

There Is a Story Behind This Object: The University Museum as a Stage

Curator: The Museum Journal, EarlyView.
ABSTRACT Objects are a critical part of most museum exhibitions and their purpose within a university museum setting is linked to the stories they carry and how these are in turn communicated to museum visitors. This paper explores these stories through the practice of museum theater, which uses performance to bring historical stories within the museum
Stephanie Jenkins
wiley   +1 more source

Rare disease clinical trials in Italy (2022-May 2026): current trends, future challenges, and the evolving role as reporting member state. [PDF]

Front Pharmacol
De Paola E, Galliccia F, Maione R, Verrelli NM, Dri DA.   +4 more
europepmc   +1 more source

Current state of medical care for patients with mycosis fungoides and Sézary syndrome at a German university hospital – a cross‐sectional study

JDDG: Journal der Deutschen Dermatologischen Gesellschaft, EarlyView.
Summary Background Studies on the state of medical care for patients with cutaneous T‐cell lymphoma (CTCL) are limited. To date, only secondary data analyses are available for Germany. Patients and Methods Cross‐sectional study conducted over a one‐year recruitment period (01 March 2024 to 28 February 2025) in the dermatological department of a German ...
Inga Hansen‐Abeck, Larissa Kölln, Finn Abeck, Glenn Geidel, Stefan W. Schneider, Nina Booken   +5 more
wiley   +1 more source

Erratum: Advancing hypoparathyroidism treatment: FDA approval of Palopegteriparatide as a promising orphan drug: Erratum. [PDF]

Ann Med Surg (Lond)
europepmc   +1 more source

International Union of Basic and Clinical Pharmacology. CXXII. Applying an objective evaluation to the status of class A orphan G protein-coupled receptors. [PDF]

Pharmacol Rev
Alexander SPH, Bennett KA, Brown AJ, Glass M, Gloriam DE, Hanson J, Insel PA, Kelly E, Langmead CJ, Martemyanov KA, Neubig RR, Offermanns S, Rosenkilde MM, Schulte G, Smith NJ, Wu H, Faccenda E, Harding SD, Davenport AP.   +18 more
europepmc   +1 more source

Clinical and genetic characterization of intellectual disability

Developmental Medicine &Child Neurology, EarlyView.
This study examines the etiological factors and comorbidities in a large cohort of Finnish patients with intellectual disability. Genetic causes—including chromosomal abnormalities and pathogenic gene variants—were more frequently identified in individuals with moderate to profound intellectual disability.
Aarni Venetvaara, Minna Kraatari‐Tiri, Jussi‐Pekka Tolonen, Marko Merikukka, IDGEN Study Group, Hannaleena Kokkonen, Teija Paakkola, Susanna Varis, Antti Dahl, Jarmo Körkkö, Jonna Komulainen‐Ebrahim, Jukka Moilanen, Johanna Uusimaa, Outi Kuismin, Elisa Rahikkala   +14 more
wiley   +1 more source