Results 51 to 60 of about 16,303 (230)

Bringing Gene Therapy Into Real World Clinical Practice

Haemophilia, EarlyView.
ABSTRACT Introduction Adeno‐associated virus (AAV)‐based gene therapy for haemophilia has shifted therapeutic paradigms by enabling hepatic gene transfer, restoring endogenous clotting factor expression, and reducing reliance on conventional prophylactic treatments. Two products, valoctocogene roxaparvovec (haemophilia A) and etranacogene dezaparvovec (
Wolfgang Miesbach, Pratima Chowdary, Margareth C. Ozelo   +2 more
wiley   +1 more source

Molecular determinants of FVIII immunogenicity in hemophilia A [PDF]

, 2013
Replacement therapy comprising regular injections with either plasma-derived or recombinant FVIII remains the major treatment used for hemophilia patients. Unfortunately, its high cost hampers its availability for many patients.
Wróblewska, A.
core   +6 more sources

FVIIIa Mimetics: New Approaches and Next‐Generation Initiatives

Haemophilia, EarlyView.
ABSTRACT Emicizumab has revolutionized hemophilia A care, yet limitations regarding the “ceiling” of hemostatic efficacy (equivalent to mild hemophilia) and global access persist. This review critically examines two distinct paradigms shaping the future of care: Innovation and Access. Regarding innovation, we synthesize the latest clinical data on next‐
Tadashi Matsushita, Keiji Nogami, Chandrakala Shanmukhaiah   +2 more
wiley   +1 more source

FREQUENCY AND LEVELS OF FACTOR EIGHT INHIBITORS IN KNOWN HAEMOPHILIACS

Pakistan Armed Forces Medical Journal, 2018
Objective: To determine the frequency and levels of factor VIII inhibitors in known haemophilics in our population. Study Design: Cross sectional study.
Mumtaz Amir, Zaheer Ul Hassan, Omer Bin Abdul Aziz, Nadir Ali, Saqib Qayyum Ahmad   +4 more
doaj  

Characterization of a factor VIII/immunoglobulin heavy chain μ double-knockout mouse model of hemophilia A for long-term exposure to factor VIII proteins

Research and Practice in Thrombosis and Haemostasis
Background: Deficiency of coagulation factor (F)VIII is the key characteristic of hemophilia A. The FVIII knockout mouse model is a valuable tool for investigating disease mechanisms and evaluating the pharmacokinetics (PK) and efficacy of therapeutic ...
Lara Monica, Olga Oleshko, Lina Aires, Silvio Wuschko, Jonas Füner, Helmut Paul, Paul Schauerte, Ditte Starberg Jespersen, Larisa Belyanskaya, Andreas Tiede, Sonja Werwitzke   +10 more
doaj   +1 more source

Gene Editing for Haemophilia—The Next Frontier

Haemophilia, EarlyView.
ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti, Gregory A. Newby, Sundar Selvaraj, Steven W. Pipe   +3 more
wiley   +1 more source

Rare Bleeding Disorders and Bleeding Disorder of Unknown Cause: Current Understanding and Recent Developments

Haemophilia, EarlyView.
ABSTRACT Rare bleeding disorders (RBDs) represent a diverse group of inherited conditions involving coagulation factors or platelets. These conditions, such as Glanzmann thrombasthenia (GT) or severe coagulation factor deficiencies, are uncommon. In contrast, bleeding disorder of unknown cause (BDUC) is a diagnosis of exclusion without an identifiable ...
Alessandro Casini, Robert Klamroth, Bauke Haisma, Dino Mehic, Saskia E. M. Schols   +4 more
wiley   +1 more source

TGA parameters for anti-fVIII MAbs.

, 2012
ETP, peak thrombin and index velocity are presented as ratios compared to fVIII deficient plasma supplemented with 1 U/ml fVIII in the absence of any anti-fVIII MAb.ETP – endogenous thrombin potential.
Bagirath Gangadharan (124016), Christopher B. Doering (124019), Shannon L. Meeks (124024), Bhavya S. Doshi (124012)   +3 more
core   +1 more source

Patient Preferences for the Treatment of Haemophilia A and B in France

Haemophilia, EarlyView.
ABSTRACT Introduction Haemophilia impacts health and quality of life. Advances in treatments such as factor replacement therapy (FRT), bispecific monoclonal antibody (BS‐mAb), gene therapy (GT), rebalancing treatment and high sustained factor necessitate understanding patient preferences to inform therapeutic strategies. Aim This study quantified trade‐
Yesim Dargaud, Yasmine Fahfouhi, Monia Ezzalfani, Astrid Foix Colonier, Alaeddine Sidhom, Aline Gauthier, Jérémie Rudant, Meriem Kessouri, Nicolas Giraud, Benoît Guillet, Anaïs Reynaud   +10 more
wiley   +1 more source

FVIII proteins with a modified immunodominant T-cell epitope exhibit reduced immunogenicity and normal FVIII activity

, 2018
Key PointsLess immunogenic FVIII muteins were designed by defining and replacing MHCII anchor residues with amino acids that reduced MHCII binding. Patient-derived T-cell clones show lower proliferation in response to FVIII-F2196K, which had normal FVIII
Joseph A. Liberman, Eddie A. James, Ruth A. Ettinger, Kathleen P. Pratt, Devi Gunasekera, Arthur R. Thompson, Komal Puranik   +6 more
core   +1 more source