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12,547 results
  • Gene therapy for hemophilia: looking beyond factor expression. [Review]
    Exp Biol Med (Maywood). 2023 Jan 23 [Online ahead of print]Yamaguti-Hayakawa GG, Ozelo MC
  • Hemophilia A (factor VIII [FVIII] deficiency) and hemophilia B (factor IX [FIX] deficiency) are the X-linked recessive bleeding disorders that clinically manifest with recurrent bleeding, predominantly into muscles and joints. In its severe presentation, when factor activity is less than 1% of normal, hemophilia presents with spontaneous musculoskeletal bleeds and may progress to debilitating chr…
  • Protection against visible light by dihydroxyacetone in erythropoietic protoporphyria. [Journal Article]
    Photodiagnosis Photodyn Ther. 2023 Jan 20 [Online ahead of print]Heerfordt IM, Philipsen PA, … Wulf HC
  • CONCLUSIONS: Both the theoretical estimates and the uncontrolled study suggest that sunless tanning with DHA reduces photosensitivity in patients with EPP. Our hypothesis is that skin treated with DHA can tolerate twice the daylight dose compared to untreated skin before onset of skin symptoms. To validate this conclusion, we plan a randomized clinical trial to determine the effect of DHA application to reduce photosensitivity in patients with EPP under controlled clinical conditions. The study protocol for this trial is presented in the paper.
  • Factor assay in victims of snake bite: Experience from a tertiary care institute in South India. [Journal Article]
    Asian J Transfus Sci. 2022 Jul-Dec; 16(2):175-179.Rafi AM, Innah SJ
  • CONCLUSIONS: Early detection and treatment of envenomation remains the cornerstone of managing snake venom-induced consumptive coagulopathy. Anti-snake venom plays a major role in the reversal of coagulopathy. Blood and blood products would be useful when coagulopathy does not revert by ASV alone. Evidence-based transfusion can be implemented and cryoprecipitate may be used as many of the patients had factor XIII and fibrinogen deficiency as part of venom-induced coagulopathy. To improve patient management and thereby the outcome of patients CMEs and training programs for the treating physicians also has to be implemented so that guidelines are formulated and followed.
  • How to translate and implement the current science of gene therapy into haemophilia care? [Review]
    Ther Adv Hematol. 2023; 14:20406207221145627.Hermans C, Gruel Y, … Krumb E
  • Gene-based therapy opens an entirely new paradigm in managing people with haemophilia (PWH), offering them the possibility of a functional cure by enabling continuous expression of factor VIII (FVIII) or factor IX (FIX) after transfer of a functional gene designed to replace the PWH's own defective gene. In recent years, significant advances in gene therapy have been made, resulting in clotting f…
  • Prothrombin Time and Coagulation Factor IX as Hemostatic Risk Markers for Legg- Calvé-Perthes Disease. [Journal Article]
    Clin Appl Thromb Hemost. 2023 Jan-Dec; 29:10760296221151166.Hernández-Zamora E, Rodríguez-Olivas AO, … Reyes-Maldonado E
  • CONCLUSIONS: Diminished prothrombin time and increase in FV activity, FVIII, FIX, and Hcy concentration support the hypothesis that microthrombi formation in small-caliber vessels could be causing avascularity and femoral necrosis, which are traits of LCPD. In addition, based on our results, we believe that the laboratory studies carried out are very useful in the diagnosis and treatment of LCPD.
  • Risk Factors for Incident CKD in Black and White Americans: The REGARDS Study. [Journal Article]
    Am J Kidney Dis. 2023 Jan 05 [Online ahead of print]Cheung KL, Crews DC, … Gutiérrez OM
  • CONCLUSIONS: Established CKD risk factors accounted for higher risk of incident CKD in Black vs. White individuals. Albuminuria was a stronger risk factor for eGFR decline and incident CKD in Black compared to White individuals. Living in the US stroke belt is a novel risk factor for CKD.
  • The development of ankylosing spondylitis in a patient with familial hemophilia. [Case Reports]
    Int J Rheum Dis. 2023 Jan 04 [Online ahead of print]Cüre O, Ilkkilic K
  • Hemophilia is a rare inherited disease which causes bleeding due to Factor VIII or Factor IX deficiency. It is usually X-linked recessive and typically affects males. Arthropathy occurs as a result of cartilage damage and chronic synovitis due to recurrent intra-articular bleeding in hemophilic patients and is mostly seen in the knee, shoulder, hip and ankle joints. There are many other diseases …
  • On the need for standardized reporting of photophysical parameters of in vitro photodynamic therapy studies. [Review]
    Photodiagnosis Photodyn Ther. 2022 Dec 29; 41:103263.Dick M, Jamal H, … Lilge L
  • In vitro dose escalation experiments are one of the first gatekeepers in therapeutic evaluation and development. This also holds for evaluating novel photosensitizers (PS) and Photodynamic Therapy (PDT) co-therapies as needed to provide dose response guidelines before engaging in further pre-clinical studies. The dose needed to achieve 50% cell kill (LD50) is a standard metric to report the poten…
  • Markers of Hereditary Thrombophilia with Unclear Significance. [Review]
    Hamostaseologie. 2022 Dec; 42(6):370-380.Sachs UJ, Kirsch-Altena A, Müller J
  • Thrombophilia leads to an increased risk of venous thromboembolism. Widely accepted risk factors for thrombophilia comprise deficiencies of protein C, protein S, and antithrombin, as well as the factor V "Leiden" mutation, the prothrombin G20210A mutation, dysfibrinogenemia, and, albeit less conclusive, increased levels of factor VIII. Besides these established markers of thrombophilia, risk fact…
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