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The Sanofi-Genzyme R&D Center[Affiliation] Publications | LitMetric

2,082 results match your criteria: "The Sanofi-Genzyme R&D Center[Affiliation]"

Background: Cancer immunotherapy has revolutionized melanoma treatment, but the high number of non-responders still emphasizes the need for improvement of therapy. One potential avenue for enhancing anti-tumor treatment is through the modulation of coagulation and platelet activity. Both have been found to play an important role in the tumor microenvironment, tumor growth and metastasis.

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Article Synopsis
  • Poor affect recognition is an early indicator of frontotemporal dementia (FTD), and this study used the Comprehensive Affect Testing System (CATS-A) to assess emotional dysfunction in FTD patients compared to healthy controls.
  • The study involved 139 FTD patients of various subtypes and tested them alongside 116 healthy controls, measuring their Affective Recognition Quotient (ARQ) to evaluate emotional recognition accuracy.
  • Results showed that the CATS-A effectively differentiated between FTD patients and healthy controls with high accuracy (AUC 0.89) and had robust internal reliability, suggesting it's a valuable clinical tool for assessing emotional dysfunction in FTD.
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Background: There has been a paradoxical rise in young-onset gastric cancer (YOGC), defined as gastric cancer (GC) diagnosed before age 50. Precursor lesions may contribute to pathogenesis, though their role in progression to different histologic subtypes is unclear. The impact of self-reported race is also poorly characterized and may be unreliable as a proxy for genetic differences.

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Design of an international, phase IV, open-label study of simoctocog alfa in women/girls with hemophilia A undergoing surgery (NuDIMENSION).

Ther Adv Hematol

December 2024

Institute of Experimental Haematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Venusberg Campus 1, Gebäude 43, Bonn 53127, Germany.

Background: Although hemophilia A mainly affects males, carriers (defined as females with hemophilia A, as well as symptomatic or asymptomatic hemophilia A carriers) are at risk of excessive bleeding, particularly during trauma or during surgical procedures. Clinical trials have focused on male patients with severe disease, and data for females are limited. Improved, evidence-based treatment guidelines for management of hemophilia A carriers are required.

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Population-Based Study of the Epidemiology of Stiff Person Syndrome in a Large Colorado-Based Health System.

Neurology

December 2024

From the Department of Neurology (P.D.C., S.S.), and Department of Biostatistics & Informatics (S.S.), University of Colorado Anschutz Medical Campus; University of Colorado School of Medicine Anschutz Medical Campus (R.D., P.W., E.E., B.V., E.M., K.V.N., A.M.C., A.L.P.); University of Colorado School of Medicine (R.F.), Aurora; School of Osteopathic Medicine (R.F.), Kanas City University, MO; University of Colorado School of Medicine Anschutz Medical Campus (R.V.C.); School of Medicine (R.V.C.), Aurora, CO; Renown Health (R.V.C.), Reno, NV; Rocky Mountain MS Center (K.V.N., A.L.P.), University of Colorado School of Medicine; and Skaggs School of Pharmacy and Pharmaceutical Sciences (K.V.N.), University of Colorado Anschutz Medical Campus, Aurora.

Article Synopsis
  • Stiff person syndrome spectrum disorder (SPSD) is a rare autoimmune condition with estimated prevalence of 1-2 cases per million, marked by muscle stiffness and painful spasms; this study aims to clarify its incidence and prevalence using data from the University of Colorado Health system.
  • A total of 273 patients were identified with potential SPSD diagnosis codes, but only 59 were confirmed to have the disorder, leading to a prevalence estimate of 2.11 cases per 100,000 persons.
  • Different clinical diagnostic criteria were assessed, revealing varying estimated prevalence rates for SPSD, indicating inconsistencies in diagnosis and classification within the population studied.
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Objectives: To assess characteristics of increased intracranial pressure (ICP) in myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD).

Methods: This is a multicenter retrospective review of 84 MOGAD cases at the University of Florida, Baylor College of Medicine, the University of California San Diego, and Providence Health and Services, Portland, Oregon, to identify cases with a documented increased opening pressure >25 cm H2O. A literature review was conducted to identify previously reported MOGAD cases with an opening pressure >25 cm H2O.

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Association between education level and access to disease-modifying treatment in patients with multiple sclerosis in France.

Mult Scler

December 2024

Univ Rennes, EHESP, CNRS, Inserm, Arènes-UMR 6051, RSMS (Recherche sur les Services et Management en Santé)-U 1309, Rennes, France.

Background: We hypothesized that differences in access to disease-modifying treatments (DMTs) could explain the association between socioeconomic status and disability progression in multiple sclerosis (MS).

Objective: This study aimed to analyze the association between education level and DMT use in France.

Methods: All patients from OFSEP network with MS onset over 1996-2014 and aged ⩾ 25 years at onset were included.

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Recently, the International Society on Thrombosis and Haemostasis (ISTH) Hemophilia Guidelines were published in this journal. The authors of these guidelines should be commended for a herculean task that took years to complete, and while this is no doubt a welcome addition to the literature, it does leave many questions for the clinician. This is primarily because 11 of the 13 recommendations are conditional, essentially meaning "that clinicians and patients need to consider individual preferences as well as the specific circumstances in which the decision is being made for implementation of the recommendation.

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Association between education level and disability progression in patients with multiple sclerosis in France.

Mult Scler

November 2024

Univ Rennes, EHESP, CNRS, Inserm, Arènes-UMR 6051, RSMS (Recherche sur les Services et Management en Santé)-U 1309, Rennes, France.

Background: Studies have reported an association between socioeconomic status and disability progression in multiple sclerosis (MS), but findings using the pre-MS individual socioeconomic status are missing.

Objective: The objective was to investigate the association between education level and disability progression.

Methods: All Observatoire Français de la Sclérose en Plaques (OFSEP) patients with MS clinical onset over 1960-2014, and aged ⩾25 years at MS onset were included.

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Spinal cord disease is important in most people with multiple sclerosis, but assessment remains less emphasized in patient care, basic and clinical research and therapeutic trials. The North American Imaging in Multiple Sclerosis Spinal Cord Interest Group was formed to determine and present the contemporary landscape of multiple sclerosis spinal cord evaluation, further existing and advanced spinal cord imaging techniques, and foster collaborative work. Important themes arose: (i) multiple sclerosis spinal cord lesions (differential diagnosis, association with clinical course); (ii) spinal cord radiological-pathological associations; (iii) 'critical' spinal cord lesions; (iv) multiple sclerosis topographical model; (v) spinal cord atrophy; and (vi) automated and special imaging techniques.

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Objective: To ascertain the changes of serum neurofilament light chain (sNfL) and glial fibrillary acidic protein (sGFAP) values in relapsing-remitting multiple sclerosis (RRMS) patients treated with ocrelizumab and their association with treatment response.

Methods: Multicenter prospective study including 115 RRMS patients initiating ocrelizumab treatment between February 2020 and March 2022 followed during a year. Serum samples were collected at baseline and every 3 months to measure sNfL and sGFAP levels using single-molecule array (SIMOA) technology.

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Article Synopsis
  • Targeted therapies using biologics for atopic diseases, urticaria, and angioedema are advancing quickly, with several new antibodies developed, tested, and approved for clinical use, like omalizumab and dupilumab.
  • There is ongoing research into combining different biologics for enhanced treatment efficacy, expanding their applications to conditions like food allergies and eosinophilic esophagitis.
  • There are emerging concerns about unexpected side effects and hypersensitivity reactions associated with these therapies, raising important questions about their safety and mechanisms, particularly in specific patient groups like children.
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Determining Line of Therapy from Real-World Data in Non-Small Cell Lung Cancer.

Pharmacoepidemiol Drug Saf

December 2024

Department of Medicine, Division of Hematology and Oncology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Article Synopsis
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Objective: Cognitive impairment affects approximately 30% of pediatric onset Multiple Sclerosis (POMS) patients with a negative impact on everyday life. The aim of this study was to evaluate the feasibility and effectiveness of a home-based, computer-assisted training of attention in patients with POMS.

Methods: This was a randomized double-blind study.

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Article Synopsis
  • The study focuses on pediatric-onset multiple sclerosis (POMS) patients, who often face cognitive issues, and examines how natalizumab (NTZ) affects their cognitive function compared to adult-onset multiple sclerosis (AOMS) patients.
  • Involving 63 patients, researchers monitored cognitive performance using the Symbol Digit Modalities Test (SDMT) over at least 24 months, noting significant cognitive improvement in both POMS and AOMS, with POMS showing greater gains.
  • Results suggest that natalizumab not only helps manage disease activity but also enhances cognitive function in POMS, making it a promising treatment option for younger patients.
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Employing effective recruitment and retention strategies to engage a diverse pediatric population in genomics research.

Am J Hum Genet

December 2024

Department of Population Health Science and Policy, Icahn School of Medicine at Mount Sinai, New York, NY, USA; Institute for Health Equity Research, Icahn School of Medicine at Mount Sinai, New York, NY, USA; Department of Medicine, Icahn School of Medicine at Mount Sinai, New York, NY, USA. Electronic address:

Article Synopsis
  • The study highlights how underrepresentation in clinical genomics research diminishes the relevance of findings and benefits, particularly emphasizing the importance of diverse participant inclusion.
  • Researchers implemented patient-centered, data-driven strategies, such as using relatable and flexible recruitment methods, to ensure a diverse sample of parents and children in a pediatric genome sequencing study.
  • As a result, a high percentage of eligible participants agreed to enroll (93.5% acceptance) and the majority of those who enrolled completed the study, demonstrating the effectiveness of engaging with underrepresented communities.
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Asthma has been increasingly recognized as a heterogeneous disease; however, many patients with asthma have allergic asthma (AA). Inhaled corticosteroids and other inhalers have been integral in treating many symptoms of asthma, but these medications do not completely address the disease's underlying mechanism. Pediatric asthma imposes a substantial burden on patients and the health care system.

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Clinical-Epidemiological Profile, and Treatment Response in Relation to Associated Atopic Comorbidity in Atopic Dermatitis. Experience From the BIOBADATOP Registry.

Actas Dermosifiliogr

November 2024

Servicio de Dermatología, Hospital Universitari Germans Trias i Pujol, Germans Trias i Pujol Research Institute (IGTP), Badalona, Barcelona, España; Departament de Medicina, Universitat Autònoma de Barcelona (UAB), Barcelona, España.

Article Synopsis
  • A study was conducted on patients with atopic dermatitis (AD) to explore the impact of atopic comorbidities like asthma and allergic rhinitis on their treatment response.
  • Data was collected from the BIOBADATOP registry, involving 509 patients, mostly adults with severe AD, and analyzed for associations between atopic comorbidities and clinical characteristics.
  • Results indicated that patients with personal atopic comorbidities had more severe symptoms and a higher history of treatments, but no significant differences in treatment response were found after 6 and 12 months, suggesting a need for longer follow-up.
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Background: Inequity in asthma and COVID-19 outcomes and research among Hispanic or Latino individuals is well established. Patient centered outcomes research (PCOR) may help address these inequalities. This report describes the development, implementation, and evaluation of a virtual conference series program titled "Unidos Hablemos de/United, Let's Talk About" intended to address the health literacy of the Hispanic/Latino community regarding asthma and COVID-19 and to encourage involvement in PCOR.

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Background: Following the pivotal phase II trial BOLT, the Hedgehog (Hh) inhibitor sonidegib was approved in the EU to treat locally advanced basal cell carcinoma (laBCC) in patients not amenable to surgery or radiotherapy. We report safety data from the interim analysis of the real-world NISSO study.

Methods: NISSO is an ongoing non-interventional, multinational, post-authorization safety study (NCT04066504).

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Article Synopsis
  • - The PROPEL trial studied the effects of cipaglucosidase alfa plus miglustat (cipa+mig) versus alglucosidase alfa plus placebo (alg+pbo) on adults with late-onset Pompe disease (LOPD) over 52 weeks, finding improvements in motor and respiratory function for those switching to cipa+mig.
  • - Patient-reported outcomes (PROs) evaluated included various measures of physical function, fatigue, and overall quality of life, with statistical analyses comparing responses between the two treatment groups.
  • - Results showed that cipa+mig significantly improved patient-reported impressions of change in ability to move around and generally outperformed alg+pbo in most PRO measures, indicating enhanced
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Purpose: Patients with relapsed or refractory (R/R) peripheral T-cell lymphoma (PTCL) generally have poor prognoses and limited treatment options. This study evaluated the efficacy of a novel CD30/CD16A bispecific innate cell engager, acimtamig (AFM13), in patients with R/R PTCL.

Patients And Methods: Patients included those with CD30 expression in ≥1% of tumor cells and who were R/R following ≥1 prior line of systemic therapy.

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