Targeting BMP signalling in cardiovascular disease and anaemia

NW Morrell, DB Bloch, P Ten Dijke… - Nature Reviews …, 2016 - nature.com
Bone morphogenetic proteins (BMPs) and their receptors, known to be essential regulators
of embryonic patterning and organogenesis, are also critical for the regulation of …

Deregulated TGF-β/BMP signaling in vascular malformations

SI Cunha, PU Magnusson, E Dejana… - Circulation …, 2017 - Am Heart Assoc
Correct organization of the vascular tree requires the balanced activities of several signaling
pathways that regulate tubulogenesis and vascular branching, elongation, and pruning …

Pazopanib may reduce bleeding in hereditary hemorrhagic telangiectasia

ME Faughnan, JR Gossage, MM Chakinala, SP Oh… - Angiogenesis, 2019 - Springer
Pazopanib (Votrient) is an orally administered tyrosine kinase inhibitor that blocks VEGF
receptors potentially serving as anti-angiogenic treatment for hereditary hemorrhagic …

Intra-venous bevacizumab in hereditary hemorrhagic telangiectasia (HHT): A retrospective study of 46 patients

A Guilhem, AE Fargeton, AC Simon, P Duffau… - PLoS …, 2017 - journals.plos.org
Background Bevacizumab, an anti-VEGF monoclonal antibody, has recently emerged as a
new option for severe forms of hereditary hemorrhagic telangiectasia (HHT). Its utilization in …

Hereditary hemorrhagic telangiectasia: the convergence of genotype, phenotype, and imaging in modern diagnosis and management of a multisystem disease

SW Hetts, JT Shieh, MA Ohliger, MB Conrad - Radiology, 2021 - pubs.rsna.org
Hereditary hemorrhagic telangiectasia (HHT) is an autosomal dominant disease that
manifests as vascular malformations in the brain, lung, liver, gastrointestinal tract, nasal …

Influence of antigen mass on the pharmacokinetics of therapeutic antibodies in humans

D Ternant, N Azzopardi, W Raoul… - Clinical …, 2019 - Springer
Therapeutic antibodies are increasingly used to treat various diseases, including neoplasms
and chronic inflammatory diseases. Antibodies exhibit complex pharmacokinetic properties …

Cerebrovascular manifestations of hereditary hemorrhagic telangiectasia

W Brinjikji, VN Iyer, T Sorenson, G Lanzino - Stroke, 2015 - Am Heart Assoc
HHT have variants of unknown significance or no detectable mutations in any of the above-
listed genes suggesting involvement of alternative pathways and mechanisms. De novo …

High output cardiac failure in 3 patients with hereditary hemorrhagic telangiectasia and hepatic vascular malformations, evaluation of treatment

LB Olsen, AD Kjeldsen, MK Poulsen, J Kjeldsen… - Orphanet Journal of …, 2020 - Springer
Background This report addresses how patients with hereditary hemorrhagic telangiectasia
(HHT) and high output cardiac failure (HOCF) due to hepatic vascular malformations, should …

Strategies for clinical development of monoclonal antibodies beyond first-in-human trials: tested doses and rationale for dose selection

M Viala, M Vinches, M Alexandre, C Mollevi… - British journal of …, 2018 - nature.com
Background: Our previous survey on first-in-human trials (FIHT) of monoclonal antibodies
(mAbs) showed that, due to their limited toxicity, the recommended phase II dose (RP2D) …

Multimodality management of complex periorbital venolymphatic malformations

S Ramesh, G Duckwiler, RA Goldberg… - Ophthalmic Plastic & …, 2019 - journals.lww.com
Purpose: To review a representative case series and share a stepwise approach to the
diagnosis and treatment of periorbital venolymphatic malformations. Methods: A case series …