Results 11 to 20 of about 20,794 (210)

Adeno‐associated virus serotype 2 capsid variants for improved liver‐directed gene therapy

Hepatology, EarlyView., 2022
Abstract Background and Aims Current liver‐directed gene therapies look for adeno‐associated virus (AAV) vectors with improved efficacy. With this background, capsid engineering is explored. Whereas shuffled capsid library screenings have resulted in potent liver targeting variants with one first vector in human clinical trials, modifying natural ...
Nadja Meumann, Marti Cabanes‐Creus, Moritz Ertelt, Renina Gale Navarro, Julie Lucifora, Qinggong Yuan, Karin Nien‐Huber, Ahmed Abdelrahman, Xuan‐Khang Vu, Liang Zhang, Ann‐Christin Franke, Christian Schmithals, Albrecht Piiper, Annabelle Vogt, Maria Gonzalez‐Carmona, Jochen T. Frueh, Evelyn Ullrich, Philip Meuleman, Steven R. Talbot, Margarete Odenthal, Michael Ott, Erhard Seifried, Clara T. Schoeder, Joachim Schwäble, Leszek Lisowski, Hildegard Büning   +25 more
wiley   +1 more source

Utilization evaluation of factor concentration and frequencyof bleeds among patients with haemophilia "A" and haemophilia "B" in northwest Iran [PDF]

Journal of Analytical Research in Clinical Medicine, 2013
Background Haemophilia A and B are X-linked bleeding disorders which result in decreased blood levels of coagulants. According to some studies, Hemophilia Severity Score (HSS) is higher in severe Haemophilia A(HA) than in severe Haemophilia B(HB ...
Roya Dolatkhah, Morteza Ghojazadeh, Iraj Asvadi Kermani, Zohreh Sanaat, Nasrin Tavassoli, Mehri Gholchin   +5 more
doaj   +1 more source

Processes and experiences of satellite haemophilia clinic set-ups in Uganda – a short report

The Journal of Haemophilia Practice, 2022
Haemophilia knowledge and care are largely missing in much of sub-Saharan Africa and there is a need for concerted efforts to ensure access to care services by affected persons. Haemophilia Foundation Uganda, supported by the global haemophilia community
Kasirye Philip, Kisakye Agnes, Nakalyango Annette, Barnabas Atwiine   +3 more
doaj   +1 more source

Health-related quality of life, direct medical and societal costs among children with moderate or severe haemophilia in Europe: multivariable models of the CHESS-PAEDs study

Orphanet Journal of Rare Diseases, 2022
Background Haemophilia bears substantial humanistic and economic burden on children and their caregivers. Characterising the differential impact of severe versus moderate paediatric haemophilia is important for clinical and health policy decisions.
Idaira Rodriguez-Santana, Pronabesh DasMahapatra, Tom Burke, Zalmai Hakimi, José Bartelt-Hofer, Jameel Nazir, Jamie O’Hara   +6 more
doaj   +1 more source

International consensus recommendations on the management of people with haemophilia B

Therapeutic Advances in Hematology, 2022
Haemophilia B is a rare X-linked genetic deficiency of coagulation factor IX (FIX) that, if untreated, can cause recurrent and disabling bleeding, potentially leading to severe arthropathy and/or life-threatening haemorrhage.
Daniel P. Hart, Davide Matino, Jan Astermark, Gerard Dolan, Roseline d’Oiron, Cédric Hermans, Victor Jiménez-Yuste, Adriana Linares, Tadashi Matsushita, Simon McRae, Margareth C. Ozelo, Sean Platton, Darrel Stafford, Robert F. Sidonio, Andreas Tiede   +14 more
doaj   +1 more source

Molecular Profiling of FIX (FACTOR 9) Gene of Hemophilia B in Karnataka

BLDE University Journal of Health Sciences, 2020
Introduction: Haemophilia is one of the “Bleeder Disorder” that causes abnormal bleeding or poor blood clotting. It occurs due to the defect in the clotting factors even in their functions and in some cases structures are altered.
Sujayendra Kulkarni, Rajat Hegde, Smita Hegde, Suyamindra Kulkarni, Sanjeev Kolagi, Pramod Gai, Rudragouda Bulagouda   +6 more
doaj   +1 more source

Three-Decade Successive Establishment of Care for Women/Girls from Families with Haemophilia

The Application of Clinical Genetics, 2022
Ampaiwan Chuansumrit,1 Werasak Sasanakul,1 Nongnuch Sirachainan,1 Suttikarn Santiwatana,1 Praguywan Kadegasem,1 Pakawan Wongwerawattanakoon,2 Noppawan Tungbubpha,1 Juthamard Chantaraamporn1 1Department of Pediatrics, Faculty of Medicine, Ramathibodi ...
Chuansumrit A, Sasanakul W, Sirachainan N, Santiwatana S, Kadegasem P, Wongwerawattanakoon P, Tungbubpha N, Chantaraamporn J   +7 more
doaj  

Treatment of a patient with severe haemophilia A presenting with left extra pleural haematoma and diagnosed with inhibitors – case report

The Journal of Haemophilia Practice, 2022
Haemophilia is an inherited X-linked bleeding disorder characterised by a deficiency or absence of clotting factor VIII (haemophilia A) or IX (haemophilia B), which can cause musculoskeletal bleeding. The standard treatment for haemophilia is with factor
Das Arijit, Dutta Anupam, BBV Ramanan, Sreeraj Sanchu TK   +3 more
doaj   +1 more source

Acquired haemophilia

Polish Archives of Internal Medicine, 2007
Acquired haemophilia (AH) is a severe bleeding diathesis that affects both males and females. It is caused by suddenly appearing autoantibodies that interfere with coagulation factor VIII (FVIII) activity. Although some conditions such as autoimmune diseases, cancer and puerperium seem likely to induce AH, in more than half of the observed cases ...
Anna, Buczma, Jerzy, Windyga
openaire   +2 more sources

Haemophilia item bank based on International Classification of Functioning, Disability and Health: a systematic review

Hematology, 2023
Background and Objectives Haemophilia comprises a continuum of blood disorders that often include joint and muscular dysfunction, which may lead to a constellation of activity limitations and participation restrictions.
Bao-Feng Geng, Chun Feng, Shou-Guo Liu, Zhong-Li Jiang, Feng Lin   +4 more
doaj   +1 more source