Results 151 to 160 of about 160,917 (252)
Clinical data required for the approval of pediatric pharmaceuticals in Japan. [PDF]
Haigo H, Matsuda K, Shikano M.
europepmc +1 more source
Abstract Background and Purpose Several G protein‐coupled receptors (GPCRs) are known to homodimerise. Dimeric GPCRs may have different properties from their monomers, but the molecular basis and functional significance of GPCR dimerisation remain largely unknown.
Satoshi Asano+6 more
wiley +1 more source
Rare disease challenges and potential actions in the Middle East. [PDF]
Fasseeh AN+11 more
europepmc +1 more source
Abstract Autoimmune diseases such as systemic lupus erythematosus (SLE) and rheumatoid arthritis (RA) result from immune self‐tolerance loss, causing tissue damage. Standard treatments targeting late‐stage inflammation often have serious side effects. Artemisinin, known for its antimalarial properties, shows potential immunomodulatory effects.
Fanchao Zhou+4 more
wiley +1 more source
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
DeepES: deep learning-based enzyme screening to identify orphan enzyme genes. [PDF]
Hirota K, Salim F, Yamada T.
europepmc +1 more source
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
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Pediatric-onset rare disease therapy pipeline yields hope for some and gaps for many: 10-year projection of approvals, treated patients, and list price revenues. [PDF]
Young CM+5 more
europepmc +1 more source
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