Publications by authors named "Masri A"

Background: For people living with Type 2 Diabetes Mellitus (T2DM), achieving optimal health outcomes requires optimal self-management and adherence to medical treatment. While some studies suggest an association between poor medication adherence and lower levels of health literacy, the evidence for this association remains inconclusive. This systematic review aimed to synthesise the evidence on the association between health literacy and medication adherence among adults from ethnic minority backgrounds living with T2DM.

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Purpose Of Review: Review advancements in therapies for transthyretin (ATTR-CM) and immunoglobulin light chain (AL-CM) cardiac amyloidosis.

Recent Findings: In ATTR-CM, tafamidis remains the cornerstone therapy, with Food and Drug Administration (FDA) approval for over 5 years. Acoramidis, another transthyretin stabilizer, has very recently been FDA-approved following positive results in the ATTRibute-CM trial.

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Background: The coexistence of transthyretin cardiac amyloidosis (ATTR-CA) and aortic stenosis (AS) is increasingly recognized, but the clinical consequences are unclear. We aimed to characterize clinical outcomes in AS plus ATTR-CA compared with only AS or ATTR-CA.

Methods And Results: In a retrospective cohort study, patients with AS only, ATTR-CA only, or AS plus ATTR-CA were identified using all-payer claims data (2015-2021).

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In the present case, a 66-year-old woman presented to the Specialty Hospital (Amman, Jordan) with recurrent post-menopausal bleeding. A pelvic ultrasound scan showed an abnormal endometrial thickness of 8 mm and no adnexal masses. An endometrial biopsy revealed abundant foamy histiocyte infiltration features suggestive of xanthogranulomatous endometritis.

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Cardiac amyloidosis represents a unique disease process characterized by amyloid fibril deposition within the myocardial extracellular space. Advances in multimodality cardiac imaging enable accurate diagnosis and facilitate prompt initiation of disease-modifying therapies. Furthermore, rapid advances in multimodality imaging have enriched understanding of the underlying pathogenesis, enhanced prognostication, and resulted in the development of imaging-based markers that reflect the amyloid burden, which is of increasing importance when assessing the response to treatment.

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: The most recent guidelines and recommendations regarding treatments of dental caries in children are shifting towards evidence-based minimal or non-invasive approaches aiming to preserve the vitality of teeth and potentially reduce the need for dental general anesthesia. This study investigated the treatment recommendations of dentists actively practicing pediatric dentistry in Germany regarding different patient cases with caries in primary teeth. The questionnaire was distributed on paper or online to pediatric dentists and general dentists practicing pediatric dentistry.

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Background: In the phase 3 randomized controlled study, ATTRibute-CM, acoramidis, a transthyretin (TTR) stabilizer, demonstrated significant efficacy on the primary endpoint. Participants with transthyretin amyloid cardiomyopathy (ATTR-CM) who completed ATTRibute-CM were invited to enroll in an open-label extension study (OLE). We report efficacy and safety data of acoramidis in participants who completed ATTRibute-CM and enrolled in the ongoing OLE.

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Article Synopsis
  • - The study aimed to identify what changes in neuropathic impairment and quality of life (QoL) are important to patients with hereditary transthyretin amyloidosis and to evaluate if the drug eplontersen provides significant improvements compared to a placebo.
  • - Researchers used data from the NEURO-TTRansform trial and various scoring systems to determine thresholds for meaningful differences, finding that eplontersen led to improvements that exceeded these thresholds in neuropathy, QoL, and nutrition measurements.
  • - Results showed eplontersen provided significant clinical benefits, suggesting that these findings could influence future clinical practices and trials regarding treatment effectiveness for patients with this condition.
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Hypertrophic cardiomyopathy (HCM) is the most common genetic heart disease, affecting 1:200 to 1:500 individuals worldwide. Guidelines on the diagnosis and management of HCM have been recently published by the European Society of Cardiology (ESC) and American societies. The ESC guidelines cover a broad range of cardiomyopathies, including HCM, with 119 recommendations, whereas the American guidelines focus exclusively on HCM with 141 specific recommendations.

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Background: Standard-of-care (SoC) medications for the treatment of obstructive hypertrophic cardiomyopathy (oHCM) are recommended as first-line therapy despite the lack of evidence from controlled clinical trials and well known off-target side effects.

Objectives: We describe the impact of SoC therapy downtitration and withdrawal in patients already receiving aficamten in FOREST-HCM (Follow-Up, Open-Label, Research Evaluation of Sustained Treatment with Aficamten in Hypertrophic Cardiomyopathy; NCT04848506).

Methods: Patients receiving SoC therapy (beta-blocker, nondihydropyridine calcium-channel blocker, and/or disopyramide) were eligible for protocol-guided SoC downtitration and withdrawal at the discretion of the investigator and after achieving a stable dose of aficamten for ≥4 weeks.

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Article Synopsis
  • Charcot-Marie-Tooth disease (CMT) is a genetic condition that impacts the peripheral nervous system.
  • A new induced pluripotent stem cell (iPSC) line, JUCTCi018-A, was developed from skin cells of a CMT2EE patient carrying a specific mutation in the MPV17 gene.
  • This iPSC line exhibits normal characteristics and pluripotency, making it a useful tool for studying the mechanisms of CMT2EE.*
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Amyloidogenic transthyretin (ATTR) amyloidosis is a relentlessly progressive disease caused by the misfolding and systemic accumulation of amyloidogenic transthyretin into amyloid fibrils. These fibrils cause diverse clinical phenotypes, mainly cardiomyopathy and/or polyneuropathy. Little is known about the aggregation of transthyretin during disease development and whether this has implications for diagnosis and treatment.

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Background: Carotenoids are present throughout retina and body its dense deposition leads to an identifiable yellow spot in the macula. Macular pigment optical density (MPOD) measured in the macula is vital to macular well-being and high-resolution visual acuity. MPOD has also been associated with various health and disease states.

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Background: Aficamten is a cardiac myosin inhibitor that mitigates left ventricular outflow gradients in obstructive hypertrophic cardiomyopathy (oHCM). The clinical efficacy of aficamten across multiple outcome domains in oHCM has not been fully defined.

Objectives: This responder analysis from the SEQUOIA-HCM (Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic oHCM) trial characterizes the clinical impact of aficamten.

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Article Synopsis
  • The SEQUOIA-HCM trial examines the effectiveness of aficamten, a new cardiac myosin inhibitor, in improving exercise capacity in adults suffering from symptomatic obstructive hypertrophic cardiomyopathy (HCM).
  • The study involves a double-blind, placebo-controlled design, with participants recruited from 101 sites across 14 countries, focusing on those with objectively measured exertional intolerance.
  • The main goal is to assess changes in integrated exercise performance after 24 weeks of treatment using a combination of peak oxygen uptake and ventilation efficiency, along with monitoring clinical health outcomes.
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Article Synopsis
  • NMOSD can occur alongside SLE, presenting with symptoms like optic neuritis and transverse myelitis; confirming the diagnosis involves testing for AQP-4 antibodies.
  • A case study of a 19-year-old female with SLE experienced vision loss and extensive spinal lesions, confirming NMOSD through MRI and CSF analysis.
  • Timely treatment is crucial to manage acute symptoms and prevent relapses, as demonstrated by the patient's severe symptoms following initial treatment.
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Background: Obstructive hypertrophic cardiomyopathy (oHCM) is characterized by left ventricular (LV) hypertrophy, LV outflow tract obstruction, and left atrial dilation, which can be associated with progressive heart failure, atrial fibrillation, and stroke. Aficamten is a next-in-class cardiac myosin inhibitor that reduces outflow tract obstruction by modulating cardiac contractility, with the potential to reverse pathological remodeling and, in turn, reduce cardiovascular events.

Objectives: This study sought to investigate the effect of aficamten on cardiac remodeling compared with placebo using cardiovascular magnetic resonance (CMR) and its association with key clinical endpoints in the SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM) CMR substudy.

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Background: Aficamten, a next-in-class cardiac myosin inhibitor, improved peak oxygen uptake (pVO) and lowered resting and Valsalva left ventricular outflow (LVOT) gradients in adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in SEQUOIA-HCM (Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic oHCM), a phase 3, multicenter, randomized, double-blinded, placebo-controlled study.

Objectives: The authors sought to evaluate the effect of aficamten on echocardiographic measures of cardiac structure and function in SEQUOIA-HCM.

Methods: Serial echocardiograms were performed over 28 weeks in patients randomized to receive placebo or aficamten in up to 4 individually titrated escalating doses (5-20 mg daily) over 24 weeks based on Valsalva LVOT gradients and left ventricular ejection fraction (LVEF).

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Background And Aims: Long-term safety and efficacy of mavacamten in patients with obstructive hypertrophic cardiomyopathy (HCM) are unknown. MAVA-LTE (NCT03723655) is an ongoing, 5-year, open-label extension study designed to evaluate the long-term effects of mavacamten.

Methods: Participants from EXPLORER-HCM (NCT03470545) could enrol in MAVA-LTE upon study completion.

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