Paroxysmal Nocturnal Hemoglobinuria (PNH)

Frequency and perturbations of various peripheral blood cell populations before and after eculizumab treatment in paroxysmal nocturnal hemoglobinuria

Read more about Frequency and perturbations of various peripheral blood cell populations before and after eculizumab treatment in paroxysmal nocturnal hemoglobinuria

While red blood cells (RBCs) and granulocytes have been more studied, platelets and reticulocytes are not commonly used in

Paroxysmal nocturnal hemoglobinuria: role of the complement system, pathogenesis, and pathophysiology

Read more about Paroxysmal nocturnal hemoglobinuria: role of the complement system, pathogenesis, and pathophysiology

The

Paroxysmal nocturnal hemoglobinuria: patient journey and burden of disease

Read more about Paroxysmal nocturnal hemoglobinuria: patient journey and burden of disease

Patients with

Paroxysmal nocturnal hemoglobinuria: current treatments and unmet needs

Read more about Paroxysmal nocturnal hemoglobinuria: current treatments and unmet needs

The current standard of care for

Clinical development of our investigational therapy, pegcetacoplan

Original Publication Date

January 8, 2021

Article Source

External Web Content

Dear members of the PNH community, We are excited to share important updates with you regarding the clinical development of our investigational therapy, pegcetacoplan, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Since our last update, we have partnered with…

Pegcetacoplan Bests Eculizumab for PNH Patients

Original Publication Date

December 28, 2020

Article Source

External Web Content

Results from the phase III PEGASUS trial suggest that the targeted C3 inhibitor pegcetacoplan improved hemoglobin levels better than eculizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH) who were still anemic despite at least three months of eculizumab therapy…

Clinical Challenges: Paroxysmal Nocturnal Hemoglobinuria — Complement inhibitors a major advance but not a cure

Original Publication Date

December 9, 2020

Article Source

External Web Content

Paroxysmal nocturnal hemoglobinuria (PNH) is an extremely rare, acquired, life-threatening disease of the blood. The name comes from one of the clinical consequences of the disease -- the breakdown of red blood cells and consequent release of hemoglobin into the urine. It often…

Paroxysmal nocturnal haemoglobinuria (PNH): novel therapies for an ancient disease

Read more about Paroxysmal nocturnal haemoglobinuria (PNH): novel therapies for an ancient disease

In the UK, early work on paroxysmal nocturnal haemoglobinuria (PNH) was conducted by John Dacie who, at the Hammersmith Hospital, first hypothesised that the PNH abnormality might arise through a somatic mutation mutation:

Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria

Read more about Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria

Paroxysmal nocturnal hemoglobinuria Paroxysmal nocturnal hemoglobinuria: (pa

One-year efficacy and safety of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy: open-label extension of a randomized study

Read more about One-year efficacy and safety of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy: open-label extension of a randomized study

Background: Ravulizumab, the only long-acting complement C5 inhibitor for adults with paroxysmal nocturnal hemoglobinuria

Subscribe to Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Frequency and perturbations of various peripheral blood cell populations before and after eculizumab treatment in paroxysmal nocturnal hemoglobinuria

Paroxysmal nocturnal hemoglobinuria: role of the complement system, pathogenesis, and pathophysiology

Paroxysmal nocturnal hemoglobinuria: patient journey and burden of disease

Paroxysmal nocturnal hemoglobinuria: current treatments and unmet needs

Clinical development of our investigational therapy, pegcetacoplan

Pegcetacoplan Bests Eculizumab for PNH Patients

Clinical Challenges: Paroxysmal Nocturnal Hemoglobinuria — Complement inhibitors a major advance but not a cure

Paroxysmal nocturnal haemoglobinuria (PNH): novel therapies for an ancient disease

Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria

One-year efficacy and safety of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy: open-label extension of a randomized study

Website Feedback

Patient Helpline