Danicopan: First Approval

C Kang - Drugs, 2024 - Springer
Danicopan (Voydeya®) is an oral complement factor D inhibitor that is being developed by
Alexion AstraZeneca Rare Disease as add-on treatment to ravulizumab or eculizumab for …

Complement‐targeted therapeutics: Are we there yet, or just getting started?

D Ricklin - European Journal of Immunology, 2024 - Wiley Online Library
Therapeutic interventions in the complement system, a key immune‐inflammatory mediator
and contributor to a broad range of clinical conditions, have long been considered important …

[PDF][PDF] Role of complement factor D in cardiovascular and metabolic diseases

Y Kong, N Wang, Z Tong, D Wang, P Wang… - Frontiers in …, 2024 - frontiersin.org
Cardiovascular and metabolic diseases (CVMDs) constitute the principal causes of death
globally. Cardiovascular diseases (CVDs) constitute more than two-thirds of the deaths …

Targeting autoimmune mechanisms by precision medicine in Myasthenia Gravis

P Cavalcante, R Mantegazza, C Antozzi - Frontiers in Immunology, 2024 - frontiersin.org
Myasthenia Gravis (MG) is a chronic disabling autoimmune disease caused by
autoantibodies to the neuromuscular junction (NMJ), characterized clinically by fluctuating …

[HTML][HTML] Therapeutic innovations for geographic atrophy: A promising horizon

EC de Oliveira Figueiredo, C Bucolo… - Current Opinion in …, 2024 - Elsevier
This mini review spotlights the most promising treatments for geographic atrophy, the
advanced form of age-related macular degeneration, often resulting in severe and …

[HTML][HTML] Navigating the Complement Pathway to Optimize PNH Treatment with Pegcetacoplan and Other Currently Approved Complement Inhibitors

P Hillmen, R Horneff, M Yeh, M Kolev… - International Journal of …, 2024 - mdpi.com
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and potentially life-threatening
hematologic disorder caused by a somatic mutation in a relevant portion of hematopoietic …

The complement model disease paroxysmal nocturnal hemoglobinuria

CQ Schmidt, B Höchsmann… - European Journal of …, 2024 - Wiley Online Library
We describe initial, current, and future aspects of complement activation and inhibition in the
rare hematological disease paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare but …

Exploring treatment strategies for paroxysmal nocturnal hemoglobinuria: an overview of registered clinical trials

VP Peixoto, C Prudêncio, M Vieira - Current Medical Research and …, 2024 - Taylor & Francis
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disease in which blood
cells lack anchored proteins that regulate the complement system. The erythrocytes are then …

Oral complement factor D inhibitor danicopan for paroxysmal nocturnal hemoglobinuria

B Xu, J Zhou - Expert Review of Clinical Pharmacology, 2024 - Taylor & Francis
Introduction Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disorder
characterized by episodic hemolysis, with additional clinical manifestations including …

Clinical characteristics and management of paroxysmal nocturnal haemoglobinuria in Latin America: a narrative review

V Goldschmidt, EI Apodaca, KM Gálvez… - Annals of …, 2024 - Springer
Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, complement-associated,
haematological disorder. The level of knowledge about the disease and its management …