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Patient-reported outcomes in newly diagnosed patients with FLT3-internal-tandem-duplication-positive acute myeloid leukaemia receiving standard chemotherapy plus quizartinib or placebo (QuANTUM-First): a global, randomised, placebo-controlled, phase 3 trial
BACKGROUND: QuANTUM-First is a randomised phase 3 trial in individuals with newly diagnosed acute myeloid leukaemia (AML) that is FLT3 internal tandem duplication (ITD) positive, showing a survival advantage for quizartinib versus placebo plus standard induction and consolidation chemotherapy with or without transplantation, followed by single-agent maintenance therapy. We evaluated the impact of quizartinib on… →
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Genetic Testing for APOL1 in Adults With Hypertension: The GUARDD-US Randomized Clinical Trial
CONCLUSIONS AND RELEVANCE: Provision of APOL1 genotype high-risk results to participants and clinicians was not associated with SBP reduction overall. Among the subset of patients with uncontrolled BP, the intervention group had a significant SBP reduction. APOL1 disclosure also increased the rate of CKD screening and diagnosis. Effects of reporting APOL1 genotype merit further investigation… →
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The feasibility of an early-years motor skill school-based intervention research project in a low-resource area of South Africa
CONCLUSION: The study highlights the feasibility of conducting intervention research in low-resource settings. It notes the challenges of using the MABC-2 due to its cost and time requirements and reports on the initial effect of the program on the motor skills of the participants. Statistical significance of positive results was affected by the small sample… →
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How to Quash Your Fear of Messing Up
If you’ve ever felt anxiety that something exciting might be happening without you, you’re familiar with fear of missing out (FOMO). But what happens when you feel an underlying worry about making a mistake? Fear of messing up (FOMU) can have the opposite effect from FOMO. Instead of spurring you to action, FOMU can lead… →
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The “SAASpocalypse”
This is an important edition addressing sentiments around a looming “SAASpocalypse.” It was first published yesterday on X and is an article written by GTMfund’s General Partner, Max Altschuler. If you’re wondering about AI’s impact on software as a whole, this is a deep dive written in Max’s first person perspective below. Let’s get into… →
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A Phase I Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Fenebrutinib and Effect on the QT/QTc Interval in Healthy Participants
Fenebrutinib is a Bruton’s tyrosine kinase inhibitor under investigation for the treatment of multiple sclerosis. The goal of this study was to investigate the effect of fenebrutinib on cardiac repolarization (QT interval) as well as its safety, tolerability, and pharmacokinetics in healthy participants. Part A was a randomized, double-blind, placebo-controlled, single-ascending dose study of therapeutic… →
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Intermittent Pneumatic Compression to Prevent Venous Thromboembolism in Critically Ill Adult Patients
CONCLUSIONS: Intermittent pneumatic compression can prevent VTE in intensive care unit patients who are at high risk of hemorrhage. →
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Overall Survival of Glioblastoma Patients Treated With a Combination of 7 Micronutrients: A Nutraceutical Trial
CONCLUSION: This study has limitations but it acts as a proof of principle towards larger studies, as clearly sufficiently powered trials are crucial in determining the nature and size of the treatment effect. Future trials should consider subgroup analysis, with respect to such factors as patient’s age at diagnosis, gender, extent of surgery, MGMT mutation… →
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Protocol for Head StART: A hybrid type II cluster randomized controlled trial evaluating community ART delivery for people newly diagnosed with HIV in refugee settlements in Uganda
BACKGROUND: Until recently, community-based antiretroviral therapy (ART) delivery was only available for people living with HIV on ART for ≥6 months with a suppressed viral load. However, early attrition from HIV care is common, particularly in refugee settlements where structural and psychosocial barriers to care are pronounced, and reducing barriers to life-saving ART sooner (e.g.,… →
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Stem cell therapy for female stress urinary incontinence: Results, limitations and lessons learned from a pilot clinical study
CONCLUSION: Autologous MSC‑based therapy for SUI was feasible and showed an acceptable short‑term safety profile in this pilot research setting; however, clinical efficacy remained modest. The manufacturing and quality control methodology is reproducible in specialized cell processing centers, but its application should remain confined to clinical research conducted in compliance with current regulations governing human… →
