The past and future of haemophilia: diagnosis, treatments, and its complications

F Peyvandi, I Garagiola, G Young - The Lancet, 2016 - thelancet.com
Haemophilia A and B are hereditary haemorrhagic disorders characterised by deficiency or
dysfunction of coagulation protein factors VIII and IX, respectively. Recurrent joint and …

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 …

Clinical severity of haemophilia A: does the classification of the 1950s still stand?

IEM Den Uijl, EP Mauser Bunschoten… - …, 2011 - Wiley Online Library
The classification of haemophilia originates from 1950s and has been adopted
unchallengedly by the ISTH in 2001. The aim of this study was: does the current …

Addressing the value of gene therapy and enhancing patient access to transformative treatments

R Salzman, F Cook, T Hunt, HL Malech, P Reilly… - Molecular Therapy, 2018 - cell.com
Although high upfront costs for the high value of gene therapy have resulted in concerns
about sufficient reimbursement to allow patient access to these therapies, the significant …

Occurrence rates of haemophilia among males in the United States based on surveillance conducted in specialized haemophilia treatment centres

JM Soucie, CH Miller, B Dupervil, B Le… - Haemophilia, 2020 - Wiley Online Library
Introduction Estimates of the size and characteristics of the US haemophilia population are
needed for healthcare planning and resource needs assessment. A network of …

Associations between intracranial haemorrhage and prescribed prophylaxis in a large cohort of haemophilia patients in the United States

C Witmer, R Presley, R Kulkarni… - British journal of …, 2011 - Wiley Online Library
Intracranial haemorrhage (ICH) is the most serious type of bleeding for patients with
haemophilia. Prior published reports regarding ICH predate the widespread provision of …

Complications of haemophilia in babies (first two years of life): a report from the Centers for Disease Control and Prevention Universal Data Collection System

R Kulkarni, RJ Presley, JM Lusher, AD Shapiro… - …, 2017 - Wiley Online Library
Aim To describe the prevalence and complications in babies≤ 2 years with haemophilia.
Methods We used a standardized collection tool to obtain consented data on eligible babies …

Intracranial haemorrhage in children and adults with haemophilia A and B: a literature review of the last 20 years

E Zanon, S Pasca - Blood Transfusion, 2018 - pmc.ncbi.nlm.nih.gov
Intracranial haemorrhage (ICH) is the most serious event in haemophiliacs, resulting in high
rates of mortality and disability. Although the use of a prophylaxis regimen has improved …

[HTML][HTML] Hemophilia a

BA Konkle - 2017 - europepmc.org
Hemophilia A is characterized by deficiency in factor VIII clotting activity that results in
prolonged oozing after injuries, tooth extractions, or surgery, and delayed or recurrent …

Women and girls with haemophilia: lessons learned

R d'Oiron, S O'Brien, AH James - Haemophilia, 2021 - Wiley Online Library
Severe and moderate factor VIII (FVIII) or IX (FIX) deficiencies in female carriers of
haemophilia are rarely observed, but mild deficiency is quite frequent, although insufficiently …