Results 51 to 60 of about 4,480,870 (245)

Re‐Purposing Sapropterin (Kuvan) for ACTA2‐Related Multisystemic Smooth Muscle Dysfunction Syndrome: A Translational Mechanistic and First‐In‐Human Therapeutic Report

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Multisystemic smooth muscle dysfunction syndrome (MSMDS) is an ultra‐rare, ACTA2‐related disorder characterized by severe cerebrovascular disease, aortic aneurysms, and smooth muscle dysfunction. Using molecular dynamics simulations and in silico drug screening, we identified that sapropterin dihydrochloride (Kuvan) is a candidate capable of ...
Moran Hausman‐Kedem, Noa Bielopolski, Vijai Krishnan, Shane Wald‐Altman, Shelly I Shrian, Omer Bar‐Yosef, Livia Kapusta, Claire Shamber, Patricia L. Musolino, Miguel Weil   +9 more
wiley   +1 more source

Melaena with Peutz-Jeghers syndrome: a case report [PDF]

, 2010
Introduction: Peutz-Jeghers syndrome (PJS) is a rare familial disorder characterised by mucocutaneous pigmentation, gastrointestinal and extragastrointestinal hamartomatous polyps and an increased risk of malignancy. Peutz-Jeghers polyps in the bowel may
Palit, Amitabh, Amitabh Palit, John R Morrissey, Jadhav, Vijay, Mathew, G. (George), Mahapatra, Sunondo R., Ernie Ruban, Morrissey, John R., Ramlal Nangalia, Vijay Jadhav, Ruban, Ernie, Bhattacharya, Sayantan, Nangalia, Ramlal, Sayantan Bhattacharya, Sunondo R Mahapatra, George Mathew   +15 more
core   +2 more sources

Discordance Between Patient and Physician Global Assessments in Early Systemic Sclerosis

Arthritis Care &Research, EarlyView.
Objective This study aims to identify factors associated with patient global assessment (PtGA) and physician global assessment (PhGA) and discordance between them in systemic sclerosis (SSc). Methods Data from adults with early SSc (<5 years) from the Collaborative National Quality and Efficacy Registry were included.
Ellen Romich, Alexis Ogdie, Alisa Stephens Shields, Peter A. Merkel, Shervin Assassi, Elana J. Bernstein, Andreea Bujor, Flavia V. Castelino, Lorinda Chung, Luke B. Evnin, Tracy M. Frech, Jessica K. Gordon, Melissa Griffith, Faye N. Hant, Monica L. Harding, Laura K. Hummers, Dinesh Khanna, Kimberly S. Lakin, Dorota J. Lebiedz‐Odrobina, Yiming Luo, Ashima Makol, Maureen D. Mayes, Zsuzsanna H. McMahan, Jerry Molitor, Duncan F. Moore, Julie Paik, Carrie Richardson, Brian Skaug, Ami A. Shah, Ankoor Shah, Virginia Steen, John M. VanBuren, Elizabeth R. Volkmann, Carleigh Zahn, Nora Sandorfi, for the CONQUER Investigators   +35 more
wiley   +1 more source

Explaining use of complementary and alternative medicine in Irritable Bowel Syndrome: a common-sense model approach [PDF]

Background: Irritable Bowel Syndrome (IBS) is a prevalent chronic functional gastrointestinal condition. Conventional medical treatment can be unsuccessful and many of those affected use complementary and alternative medicine (CAM) for symptom relief ...
Usher, Lee, Fox, Pauline, Mitchell, Kathryn   +2 more
core   +3 more sources

The Gut–Heart Axis in Systemic Sclerosis: Evidence From a Large Prospective Early Disease Cohort

Arthritis Care &Research, EarlyView.
Objective Cardiac involvement significantly impacts prognosis in systemic sclerosis (SSc), highlighting the need for early risk stratification. Gastrointestinal (GI) symptoms are common and often manifest early. Emerging data suggest a link between GI and cardiac manifestations, possibly through shared mechanisms like dysautonomia.
Francesca R. Di Ciommo, Ashish P. Balar, Aidan K. Strother, Subhash Kulkarni, Michael Hughes, Brian Skaug, Maureen D. Mayes, Shervin Assassi, Ali Y. Ayla, Zsuzsanna H. McMahan   +9 more
wiley   +1 more source

Incidence, Risk Factors and Management of Adverse Events in Contemporary Real‐World Care of Children with Juvenile Idiopathic Arthritis

Arthritis Care &Research, Accepted Article.
Objective We describe the frequency, risk factors, severity, and management of actionable and serious adverse events (AAE and SAE) in children with newly diagnosed Juvenile Idiopathic Arthritis (JIA) in Canada. Methods We enrolled patients within 3 months of JIA diagnosis in the Canadian Alliance of Pediatric Rheumatology Investigators (CAPRI) Registry,
Bashayer Alnuaimi, Roberta A. Berard, Jean‐Philippe Proulx‐Gauthier, Heinrike Schmeling, Michelle Batthish, Gilles Boire, Adam M. Huber, Dax G. Rumsey, Jaime Guzman, Gaëlle Chédeville, The CAPRI registry Investigators   +10 more
wiley   +1 more source

Advanced Materials for Biologics Delivery to Brain Tumors

Advanced Materials, EarlyView.
Material innovation is central to unlocking the therapeutic potential of biologics against many central nervous system diseases, including brain cancer. By engineering carriers with controlled transport, targeting, and release properties, advanced materials can overcome the blood–brain barrier and tumor microenvironment, improving the delivery of ...
Yuran Feng, Yuxue Cao, Maria Kavallaris, Taskeen Janjua, Amirali Popat   +4 more
wiley   +1 more source

Citrullinemia stimulation test in the evaluation of the intestinal function

Nutrición Hospitalaria, 2013
Background: Citrullinemia is been reported as a quantitative parameter of the enterocyte mass and function. Aim: The objective of this research is to analyse the value of fasting and stimulated citrullinemias in the intestinal function evaluation ...
Beatriz Pinto Costa, Marco Serôdio, Marta Simões, Caria Veríssimo, F. Castro Sousa, Manuela Grazina   +5 more
doaj   +1 more source

Region‐Specific Profiling of the Gut Microbiome and Metabolite Landscape in Response to Dietary Intervention Using Electronics‐Free Ingestible Smart Sampling Capsules

Advanced Materials Technologies, EarlyView.
Region‐specific profiling of the gut microbiome is achieved non‐invasively using electronics‐free ingestible sampling capsules that activate at defined intestinal sites through stimuli‐responsive coatings. Deployed in a porcine dietary intervention model, the capsules resolve spatially distinct, fiber‐driven shifts in microbial composition and short ...
Devendra Sarnaik, Caitlyn Sullivan, Akshay Krishnakumar, Fangxin Chen, Anna Clapp Organski, Wayne W. Campbell, Jay S. Johnson, Tzu‐Wen L. Cross, Rahim Rahimi   +8 more
wiley   +1 more source

The Influence of Neocate in Paediatric Short Bowel Syndrome on PN Weaning

Journal of Nutrition and Metabolism, 2010
Clinical management of short bowel syndrome remains a multistage process. Although PN is crucial, early introduction of enteral feeding is mandatory. We describe retrospectively 4 patients with an ultrashort bowel who could be weaned off PN on very short
E. De Greef, T. Mahler, A. Janssen, H. Cuypers, G. Veereman-Wauters   +4 more
doaj   +1 more source