Results 81 to 90 of about 10,277 (199)

Novel Therapeutics for Chronic Obstructive Pulmonary Disease: From Empirical Bronchodilation to Precision Medicine

open access: yesRespirology, EarlyView.
ABSTRACT Chronic obstructive pulmonary disease (COPD) remains the third leading cause of death worldwide, and conventional bronchodilator‐based therapies have limited efficacy in preventing exacerbations and disease progression. The 2024–2026 period represents a historic inflection point: three mechanistically distinct agents received Food and Drug ...
Naoya Fujino, Hisatoshi Sugiura
wiley   +1 more source

Vasopressin regulates the growth of the biliary epithelium in polycystic liver disease [PDF]

open access: yes, 2016
The neurohypophysial hormone arginine vasopressin (AVP) acts by three distinct receptor subtypes: V1a, V1b, and V2. In the liver, AVP is involved in ureogenesis, glycogenolysis, neoglucogenesis and regeneration. No data exist about the presence of AVP in
Alpini, Gianfranco   +11 more
core   +1 more source

Update on Non‐Biological and RNA‐Based Therapeutics in Chronic Inflammatory Diseases: Precision Medicine Through Small Molecules: An EAACI Position Paper

open access: yesAllergy, EarlyView.
ABSTRACT In the last decades, critical advancements in research technology and knowledge on disease mechanisms steered therapeutic approaches for chronic inflammatory diseases towards unprecedented target specificity. For allergic and chronic lung diseases, biologic drugs pioneered this goal, acquiring on the way—through the clinical use of monoclonal ...
F. Roth‐Walter   +20 more
wiley   +1 more source

Pregnancies in women with rare diseases: Selected maternal and perinatal outcomes

open access: yesActa Obstetricia et Gynecologica Scandinavica, EarlyView.
Pregnancies in women with rare diseases carry substantial disease‐related (23.2%) and pregnancy‐specific (25.1%) risks. Complication rates drop markedly when conditions are stable before conception. Abstract Introduction Rare diseases (RD) are characterized by chronicity and may be associated with reduced life expectancy and quality of life.
Philipp Kosian   +6 more
wiley   +1 more source

Adenine base editing with engineered virus-like particles rescues the CFTR mutation G542X in patient-derived intestinal organoids

open access: yesiScience
Summary: Cystic fibrosis (CF) is a life-shortening autosomal recessive disease, caused by loss-of-function mutations that affect the CF transmembrane conductance regulator (CFTR) anion channel.
Lucia Nicosia   +13 more
doaj   +1 more source

Delayed diagnosis of cystic fibrosis associated with R117H on a background of 7T polythymidine tract at intron 8 [PDF]

open access: yes, 1933
We report late diagnoses of cystic fibrosis (CF) in two men aged 61 and 65 years. At the time of presentation, both patients had significant pulmonary disease.
D. Peckham   +17 more
core   +1 more source

The potential for biased signalling in the P2Y receptor family of GPCRs

open access: yesBritish Journal of Pharmacology, EarlyView.
The purinergic receptor family is primarily activated by nucleotides, and contains members of both the G protein coupled‐receptor (GPCR) superfamily (P1 and P2Y) and ligand‐gated ion channels (P2X). The P2Y receptors are widely expressed in the human body, and given the ubiquitous nature of nucleotides, purinergic signalling is involved with a plethora
Claudia M. Sisk   +2 more
wiley   +1 more source

CFTR modulators response of S737F and T465N CFTR variants on patient-derived rectal organoids

open access: yesOrphanet Journal of Rare Diseases
Background Predictions based on patient-derived materials of CFTR modulators efficacy have been performed lately in patient-derived cells, extending FDA-approved drugs for CF patients harboring rare variants.
Karina Kleinfelder   +6 more
doaj   +1 more source

Mechanisms of lipid malabsorption in Cystic Fibrosis: the impact of essential fatty acids deficiency [PDF]

open access: yes, 2005
Affiliation: CHU-Sainte-Justine, Université de MontréalTransport mechanisms, whereby alimentary lipids are digested and packaged into small emulsion particles that enter intestinal cells to be translocated to the plasma in the form of chylomicrons, are ...
Drouin, Éric   +3 more
core   +2 more sources

Differential segment‐specific signalling pathways for guanylate cyclase C‐activated anion secretion in murine ileocolon

open access: yesBritish Journal of Pharmacology, EarlyView.
Abstract Background and Purpose Guanylate cyclase‐C (GC‐C) is the receptor for endogenous (uro)guanylin peptides, bacterial toxins and pharmacological analogues. Receptor activation leads to intestinal fluid loss, but also activates an antiproliferative pathway and is a promising target in colorectal cancer therapy.
Renjie Xiu   +4 more
wiley   +1 more source

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