147 research outputs found

    RESTORE Survey on the Public Perception of Advanced Therapies and ATMPs in Europe — Why the European Union Should Invest More!

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    Advanced therapy medicinal products (ATMPs) are potential game changers in modern medical care with an anticipated major impact for patients and society. They are a new drug class often referred to as "living drugs," and are based on complex components such as vectors, cells and even tissues. The production of such ATMPs involves innovative biotechnological methods. In this survey, we have assessed the perception of European citizens regarding ATMPs and health care in Europe, in relation to other important topics, such as safety and security, data protection, climate friendly energy supply, migration, and others. A crucial question was to determine to what extent European citizens wish to support public funding of innovations in healthcare and reimbursement strategies for ATMPs. To answer this, we conducted an online survey in 13 European countries (representative of 85.3% of the entire EU population including the UK in 2020), surveying a total of 7,062 European citizens. The survey was representative with respect to adult age groups and gender in each country. Healthcare had the highest ranking among important societal topics. We found that 83% of the surveyed EU citizens were in support of more public funding of technologies in the field of ATMPs. Interestingly, 74% of respondents are in support of cross-border healthcare for patients with rare diseases to receive ATMP treatments and 61% support the reimbursement of very expensive ATMPs within the European health care system despite the current lack of long-term efficacy data. In conclusion, healthcare is a top ranking issue for European Citizens, who additionally support funding of new technologies to enable the wider application of ATMPs in Europe

    Holt Oram syndrome: a case report and review of the literature

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    Holt Oram syndrome is a rare autosomal dominant syndrome on average, of varying severity, which may result in heterogeneous pictures, predominantly with involvement of the bony segments of the upper limbs and the cardiovascular system. The syndrome is caused by mutations in two genes of the T-box (TBX5, 601 620 and TBX 3) located on the 12q24.1p. The authors report a case and review the literature

    Rectovaginal endometriosis: head to head comparison between sonovaginography and magnetic resonance imaging

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    Background: Rectovaginal endometriosis (RVE) is a severe form of deep pelvic endometriosis associated with dysmenorrhoea, pelvic pain, and dyspareunia. Diagnosis of RVE is a challenge for clinicians. Aim of outhe present study was to compare the diagnostic accuracy of sonovaginography with MRI on a consecutive cohort of women referred to this institution due to RVE suspect. Materials and Methods: The authors performed a retrospective study on consecutive patients undergoing surgery at this Unit due to a suspicion of RVE. All women were subject to MRI and sonovaginography. Primary endpoint was to compare the diagnostic accuracy of sonovaginography and MRI in the detection of RVE. Results: RVE was diagnosed (with surgery plus histology) in 60 women. In all cases, the surgical procedure was completed without complications. Sonovaginography and MRI showed high sensitivity (95% vs. 81.7%) and similar specificity (93.8% and 91.2%, respectively) in the diagnosis of RVE. Conclusions: Sonovaginography, when performed by an expert sonographer, may represent a valid alternative to MRI for the diagnosis of RVE, with lower costs and minimal time consumption

    Accuracy of transvaginal ultrasound, saline infusion sonohysterography, and office hysteroscopy in the diagnosis of endometrial polyps

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    Background: Endometrial polyps (EPs) derive from overgrowths of stromal or endometrial glands supported by a fibrovascular core. Currently, there is no clear recommendation on the first-line tool to be employed between sonohysterography (SIS) and office hysteroscopy (ISC) in the evaluation of patients with the suspect of EPs. Materials and Methods: The authors performed a retrospective study on consecutive patients referred at their Unit with a suspicion of EP at transvaginal sonography (TVS). A total number of 1,243 patients were subject to TVS and ISC, while 128 ones were subject to TVS, SIS, and ISC. Primary outcome was the evaluation of the diagnostic performance of SIS and ISC in the detection of EPs. Results: ISC allowed the identification of EPs in all the cases (n=128/128 patients), with a sensitivity of 100% and specificity of 98%. For SIS, the sensitivity was 97% (n=124/128 patients) and specificity was 90%. Conclusions: ISC showed better diagnostic accuracy in comparison to SIS. Due to high diagnostic accuracy and the possibility to \u201csee and treat\u201d EPs in a single step, ISC should be considered as the gold standard approach in women with a suspect of EPs
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