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  1. 343261
    “…If confirmed in future studies, these findings may have implications for patient stratification, outcome measure development, and design of clinical and pre-clinical trials in FXS. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1186/s13229-019-0271-7) contains supplementary material, which is available to authorized users.…”
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    Incomplete silencing of full mutation alleles in males with fragile X syndrome is associated with autistic features
  2. 343262
    por Chao, Dyna YP, Lin, Tom MY, Ma, Wen-Ya
    Publicado 2019
    “…An experimental design (n=121) and randomized controlled trials were employed to determine patient preferences in the health promotion program (n=62) and mobile self-management education (n=28). …”
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    Enhanced Self-Efficacy and Behavioral Changes Among Patients With Diabetes: Cloud-Based Mobile Health Platform and Mobile App Service
  3. 343263
    “…STUDY REGISTRATION: ClinicalTrials.gov NCT03335644.…”
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    Ultra-processed food intake and risk of cardiovascular disease: prospective cohort study (NutriNet-Santé)
  4. 343264
    “…BACKGROUND: Network meta-analyses (NMAs) provide comparative treatment effects estimates in the absence of head-to-head randomized controlled trials (RCTs). This NMA compared the efficacy and safety of dolutegravir (DTG) with other recommended or commonly used core antiretroviral agents. …”
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    Comparative efficacy and safety of dolutegravir relative to common core agents in treatment-naïve patients infected with HIV-1: a systematic review and network meta-analysis
  5. 343265
    “…Observing their progressive accumulation in the brains of clinically normal older adults is critically important to understand the pathophysiologic cascade leading to AD and to inform the choice of outcome measures in prevention trials. OBJECTIVE: To assess the associations among Aβ, tau, and cognition, measured during different observation periods for 7 years. …”
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    Association of Amyloid and Tau With Cognition in Preclinical Alzheimer Disease: A Longitudinal Study
  6. 343266
    “…While many autoimmune diseases are known to be more prevalent in certain racial groups, it is unknown if there are racial disparities in the rate of irAEs. Clinical trials reporting irAEs thus far have not explored their distribution in underrepresented minorities. …”
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    MON-603 Racial Distribution of Endocrine Complications in Oncology Patients Treated with Immune Checkpoint Inhibitors
  7. 343267
    “…The calcimimetic drug, cinacalcet, has been trialed for treatment of FHH3 with more than 20% reduction in serum calcium levels and improvement of symptoms (2).Conclusion: FHH3 is a rare cause of hypercalcemia. …”
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    SAT-506 Identification of a Mutation in AP2S1 Causing Familial Hypocalciuric Hypercalcemia Type 3 in a Patient with Unknown Family History of the Disease
  8. 343268
    “…The JAK family kinases are targeted in cancers due to their role in cytokine receptor signal transduction, and are also key intermediates in growth hormone, leptin and prolactin receptor signaling. Clinical trials have reported that ruxolitinib treatment was associated with weight gain, but other metabolic consequences remain unknown. …”
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    SAT-091 Metabolic Effects of JAK1/2 Inhibition in Patients with Myeloproliferative Neoplasms
  9. 343269
    “…Genetic testing revealed a heterozygous mutation in the insulin receptor gene for a missense variant pointing towards the diagnosis of Type A IR.The patient had been prescribed treatment with metformin and spironolactone although her adherence to taking her medications was sporadic. She was then trialed on an oral contraceptive which led to monthly menstrual cycles. …”
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    SAT-291 Gonadotropin Releasing Hormone Agonist Therapy Improves Hyperandrogenism in an Adolescent with Type A Insulin Resistance Syndrome
  10. 343270
    “…The goals of this study are to 1) estimate and define the clinical spectrum of rare cancers, 2) evaluate and follow biological relatives of patients with rare tumors or carriers of germline genetic variants that predispose to development of rare tumors, and 3) develop a better understanding of these diseases in an effort to a) develop novel therapeutic interventions, b) preventive/screening guidelines, c) endpoints for future clinical trials, and d) identify relevant patient reported outcomes that can improve our understanding of patients’ psychosocial and functional needs. …”
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    SUN-341 A Natural History Study for Rare Tumors: An Avenue for Discovering and Understanding Endocrine Cancers
  11. 343271
    “…The aim of this prospective study is the feasibility testing of targeting CCK-2 receptors with the novel gastrin analogue [(177)Lu-DOTA-(DGlu)6-Ala-Tyr-Gly-Trp-Nleu-Asp-PheNH2] ((177)Lu-PP-F11N) in six patients with advanced MTC (ClinicalTrials.gov: NCT02088645). Subjects and Methods: Six patients received two injections of 1 GBq (177)Lu-PP-F11N, one injection with and the other without Physiogel (Gelofusin = plasma expander for nephroprotection) infusion. …”
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    SUN-334 Evaluation of the CCK-2 Receptor Agonist (177)Lu-PP-F11N for PRRT of Medullary Thyroid Carcinoma: Results of a Phase 0 "Lumed" Study
  12. 343272
    por Qureshi, Mohammed
    Publicado 2019
    “…This is recognized to be a rare immune-related endocrinopathy secondary to Pembrolizumab, with DKA occurring in only 0.1% of patients in clinical trials. Pembrolizumab blocks PD-1 receptors on the surface of T cells in order to restore T cell anti-tumor function. …”
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    SUN-172 Diabetic Ketoacidosis Precipitated by Treatment with PD-1 Inhibitor: A Rare Immune Related Adverse Event
  13. 343273
    “…Burosumab is an anti-FGF23 fully human monoclonal antibody, and the first treatment to target the underlying pathophysiology of XLH. The trials that formed the basis of regulatory approval of burosumab only included patients up to the age of 12 years old and so data are lacking on this important patient population. …”
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    SUN-525 Burosumab Experience In A UK Adolescent Population
  14. 343274
    “…RESULTS: Of 5848 articles retrieved, 24 randomized clinical trials (825 participants) were included. Among the included studies, 92% reported to have performed a randomization process, 8% presented allocation concealment, 21% reported blinding of outcome assessment, and 38% reported complete outcome data. …”
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    The Role of Aerobic Training Variables Progression on Glycemic Control of Patients with Type 2 Diabetes: a Systematic Review with Meta-analysis
  15. 343275
    “…The study outcomes will contribute refining service workflows, improving health risk assessment and generating recommendations for service selection. TRIALS REGISTRATION: NCT03130283 (date released 04/06/2018), NCT03768050 (date released 12/05/2018), NCT03767387 (date released 12/05/2018). …”
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    Evaluation of integrated care services in Catalonia: population-based and service-based real-life deployment protocols
  16. 343276
    “…Hence, the new QLQ-AA/PNH with 54 items can be used in trials and clinical studies from now on, according to EORTC strategy even if the scoring algorithm at this point is preliminary and the QLQ-AA/PNH might change slightly after phase IV. …”
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    Design and development of a disease-specific quality of life tool for patients with aplastic anaemia and/or paroxysmal nocturnal haemoglobinuria (QLQ-AA/PNH)—a report on phase III
  17. 343277
    “…Clinical studies are ongoing to evaluate immune checkpoint inhibitors (ICI) in association with RT. However, only few trials aim to optimize the RT fractionation to improve efficacy of these associations. …”
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    Optimized fractionated radiotherapy with anti-PD-L1 and anti-TIGIT: a promising new combination
  18. 343278
    “…REGISTRATION: NCT02760784 (ClinicalTrials.gov).…”
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    Gait Function and Postural Control 4.5 Years After Nonoperative Dynamic Treatment of Acute Achilles Tendon Ruptures
  19. 343279
    “…RESULTS: Nine randomized trials (n = 1437) and 5 retrospective studies (n = 421) comparing EN feeding tubes and PN were deemed eligible for the pooled analyses, with a categorized time frame of PODs ≥ 7 and PODs < 7. …”
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    Short-term evaluation of immune levels and nutritional values of EN versus PN in gastric cancer: a systematic review and a meta-analysis
  20. 343280
    “…Patients and methods: We enrolled mTNBC patients with lung metastasis who underwent (18)F-FDG PET/CT scans before first‐line therapy from three clinical trials (NCT01287624, NCT02341911 and NCT02546934). …”
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    Heterogeneity of targeted lung lesion predicts platinum-based first-line therapy outcomes and overall survival for metastatic triple-negative breast cancer patients with lung metastasis: a “PET biopsy” method
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