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Safety and efficacy of fordadistrogene movaparvovec in ambulatory participants with Duchenne muscular dystrophy (CIFFREO): a phase 3, double-blind, randomised, placebo-controlled study
BACKGROUND: Gene transfer is a promising therapeutic approach for Duchenne muscular dystrophy as the disease results from mutations in a single gene. Fordadistrogene movaparvovec is an investigational recombinant adeno-associated virus 9 (rAAV9)-based vector encoding a mini-dystrophin transgene protein for Duchenne muscular dystrophy . We aimed to assess the safety and efficacy of fordadistrogene movaparvovec in… →
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Home-based extended rehabilitation for older people with frailty (HERO): a randomised controlled trial
CONCLUSIONS: We do not recommend routine commissioning of extended rehabilitation for older people with frailty after discharge home from hospital or intermediate care, following an acute admission with a medical illness or injury. →
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How to Price Your Product: A Pricing Model Decision Tree
Recently, we published an edition called How to Price Your AI Product, co-written with DealOps, that went through critical considerations for pricing. The demand that we got for more pricing information was overwhelmingly positive. We wrote this edition to answer that call and build out a true pricing model decision tree, along with a full… →
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Single-Entry-Point Thread Implantation Technique Using the Gerbera Pattern for Facial Skin Rejuvenation: A Prospective Intraindividual Controlled Clinical Study
CONCLUSION: The single-entry-point thread implantation technique using the Gerbera pattern significantly reduced pain at needle penetration compared with the conventional multiple-entry-point technique, demonstrating good tolerability in clinical practice. →
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Sex- and Age-Stratified Outcomes of Colonoscopy Versus Faecal Immunochemical Testing: Post-Analysis of the COLONPREV Study
CONCLUSION: Participation and reductions in CRC-related mortality and incidence were consistent across sex and age groups for both FIT- and colonoscopy-based screening strategies. The higher baseline risk observed in men could not be fully mitigated by screening. →
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Cost and cost-effectiveness of ivermectin mass drug administration for malaria control in Kwale county, Kenya: a modelling analysis of a cluster-randomised trial
BACKGROUND: Malaria remains a major health burden in sub-Saharan Africa, where traditional vector control methods are hindered by insecticide resistance and evolving mosquito behaviour causing residual transmission. In the BOHEMIA cluster-randomised trial in Kenya, ivermectin mass drug administration (iMDA), delivered once a month for 3 months with approximately 64% population coverage, was shown to reduce… →
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A Randomized Clinical Trial: The Comparative Therapeutic Efficacy of Tap Water-UVA, Saltwater-UVA, and Conventional Bath PUVA in Palmoplantar Pustulosis
CONCLUSIONS: Immersion in water or saltwater prior to UVA without psoralens may provide a therapeutic effect. →
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Cost-effectiveness of transdiagnostic group cognitive behavioral therapy for emotional disorders in primary care: An analysis of the PsicAP clinical trial
CONCLUSIONS: The integration of TD-GCBT into TAU represents a cost-effective therapeutic approach. The incremental cost-effectiveness ratio of 4,759€ per quality-adjusted life years is well below the willingness-to-pay threshold accepted in Spain, which ranges from 20,000€ to 25,000€. At this threshold, the probability that the intervention is cost-effective is high, ranging from 86% to 89%. Therefore,… →
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Ask Sanyin: What Makes a ‘Listening Tour’ Meaningful?
Carolyn Geason-Beissel/MIT SMR | Getty Images I’ve just stepped into a new leadership role and was advised to embark on a “listening tour.” I’ve gone out and spent time with different units and functions, but I’m not sure it’s helping. Conversations feel surface-level, and I’m not learning what I need to lead effectively. What am… →