Haemophilia

E Berntorp, K Fischer, DP Hart, ME Mancuso… - Nature reviews Disease …, 2021 - nature.com
Haemophilia A and B are rare congenital, recessive X-linked disorders caused by lack or
deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively. The severity of the disease …

Antibody structure and function: the basis for engineering therapeutics

ML Chiu, DR Goulet, A Teplyakov, GL Gilliland - Antibodies, 2019 - mdpi.com
Antibodies and antibody-derived macromolecules have established themselves as the
mainstay in protein-based therapeutic molecules (biologics). Our knowledge of the structure …

The dawn of a new era: targeting the “undruggables” with antibody-based therapeutics

L Qian, X Lin, X Gao, RU Khan, JY Liao, S Du… - Chemical …, 2023 - ACS Publications
The high selectivity and affinity of antibodies toward their antigens have made them a highly
valuable tool in disease therapy, diagnosis, and basic research. A plethora of chemical and …

The changing treatment landscape in haemophilia: from standard half-life clotting factor concentrates to gene editing

ME Mancuso, JN Mahlangu, SW Pipe - The Lancet, 2021 - thelancet.com
Congenital haemophilia A (factor VIII deficiency) and B (factor IX deficiency) are X-linked
bleeding disorders. Replacement therapy has been the cornerstone of the management of …

[HTML][HTML] Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran—Results of the phase 1 inhibitor cohort

KJ Pasi, T Lissitchkov, V Mamonov, T Mant… - Journal of Thrombosis …, 2021 - Elsevier
Background Fitusiran, an investigational small interfering RNA therapy, reduces
antithrombin production to rebalance hemostasis in people with hemophilia A or B, with or …

New therapies for hemophilia

AC Weyand, SW Pipe - Blood, The Journal of the American …, 2019 - ashpublications.org
Hemophilia A (HA) and hemophilia B (HB) are the most common severe bleeding disorders.
Replacement therapy, providing the missing coagulation factor, has been the mainstay of …

[HTML][HTML] Factor VIII replacement is still the standard of care in haemophilia A

L Aledort, PM Mannucci, W Schramm… - Blood …, 2019 - ncbi.nlm.nih.gov
Prophylactic factor VIII (FVIII) has dramatically improved haemophilia A treatment,
preventing joint bleeding and halting the deterioration of joint status. FVIII products with an …

Pharmacokinetics and pharmacodynamics of emicizumab in persons with hemophilia A with factor VIII inhibitors: HAVEN 1 study

C Schmitt, JI Adamkewicz, J Xu, C Petry… - Thrombosis and …, 2021 - thieme-connect.com
Emicizumab, a bispecific monoclonal antibody, bridges activated factor IX (FIXa) and FX,
replacing the function of missing FVIIIa to restore effective hemostasis in persons with …

Targeting the liver with nucleic acid therapeutics for the treatment of systemic diseases of liver origin

A Gogate, J Belcourt, M Shah, AZ Wang… - Pharmacological …, 2024 - Elsevier
Systemic diseases of liver origin (SDLO) are complex diseases in multiple organ systems,
such as cardiovascular, musculoskeletal, endocrine, renal, respiratory, and sensory organ …

Effects and interferences of emicizumab, a humanised bispecific antibody mimicking activated factor VIII cofactor function, on coagulation assays

JI Adamkewicz, DC Chen… - Thrombosis and …, 2019 - thieme-connect.com
Emicizumab bridges activated factor IX (FIX) and FX to restore the tenase function mediated
by activated FVIII (FVIIIa), which is deficient in people with haemophilia A (PwHA). Unlike …