Results 291 to 300 of about 3,868,024 (353)
Clinical &Experimental Allergy, Volume 55, Issue 8, Page 659-670, August 2025.Through the ‘4As’: Awareness, Access, Advocacy, and Alliance, the Greater Bay Area will continue to leverage its unique advantages to confront ongoing challenges such as diagnostic delays and lack of access to diagnosis and hereditary angioedema (HAE)‐specific medications.
Philip H. Li +22 more
wiley +1 more source
JDDG: Journal der Deutschen Dermatologischen Gesellschaft, Volume 23, Issue 8, Page 968-987, August 2025.Summary Vitiligo is a common pigment disorder of the skin resulting in destruction of melanocytes. Non‐segmental vitiligo (NSV) is an autoimmune disorder. The etiopathogenesis of segmental vitiligo (SV) remains incompletely understood. Genetic predisposition and increased vulnerability of melanocytes towards stressors lead to a melanocyte‐specific CD8+
Markus Böhm, Adrian Tanew
wiley +1 more source
What is known about the cost-effectiveness of orphan drugs? Evidence from cost-utility analyses
, 2015 Eline Picavet +2 more
openalex +1 more source
European Journal of Haematology, Volume 115, Issue 2, Page 125-133, August 2025.ABSTRACT Background Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra‐rare, acquired non‐malignant haematological disorder characterised by thrombosis risk, serious complications and debilitating symptoms in untreated patients. Objective This anchored indirect treatment comparison (ITC) evaluated efficacy data between proximal complement 3 ...
Regis Peffault de Latour +9 more
wiley +1 more source
Orphan drugs for sickle vaso-occlusion: dawn of a new era of targeted treatment
, 2015 Carlton Dampier
openalex +2 more sources
European Journal of Haematology, Volume 115, Issue 2, Page 134-141, August 2025.ABSTRACT Objective To report data from an ITI chart review study (NCT03951103) for first‐time and rescue ITI with recombinant factor VIII Fc fusion protein (rFVIIIFc) in persons with haemophilia A. Methods Retrospective and prospective real‐world data are reported from a non‐interventional, multicentre study of patients who had been or were currently ...
Robert Klamroth +7 more
wiley +1 more source
Pharmacology Research &Perspectives, Volume 13, Issue 4, August 2025.
Ida Štimac +3 more
wiley +1 more source
Histopathology, Volume 87, Issue 2, Page 223-234, August 2025.ROS1 antibody clones are not interchangeable in the breast cancer setting and highlight major discordances in ROS1 status according to antibody clone utilised. Aims Invasive lobular carcinoma (ILC) may show targetable vulnerabilities secondary to the characteristic loss of the cell adhesion protein E‐cadherin.
Anna Sokolova +9 more
wiley +1 more source