The Canadian Association of PNH Patients is a not-for-profit Canadian organization formed in 2009. The mission of the organization is to connect Canadians affected by Paroxysmal Nocturnal Haemoglobinuria and advocate for the best possible care for patients and ensure they are equipped with the most current tools and information to help them live well with the condition. The organization also provides support to caregivers, and works to increase awareness and understanding of PNH.
Paroxysmal Nocturnal Haemoglobinuria (PNH) is a very rare and debilitating disease of the bone marrow that affects the blood and major organs. It is a chronic, life-threatening illness and can have devastating effects upon a patient physically, mentally and emotionally. This website represents a growing community of patients, caregivers and members of the medical community who wish to support and improve the quality of life of those with PNH.
FREE APP! Manage your PNH journey from anywhere in the world with Backpack Health.The Canadian Association of PNH Patients has partnered with Backpack Health, LLC to provide a free, secure health data management platform to help individuals, families and caregivers in the PNH community manage their medical information and gain better access to patient-specific resources. The Backpack Health platform will also serve as the first patient-led disease registry in PNH anywhere in the world contributing to important research advances. Learn more about the Anytime Access to PNH Health group today!
Apellis Reports Positive Results from Phase 3 Study Comparing Pegcetacoplan (APL-2) to Eculizumab (Soliris) in PNH Patients
January 16, 2020
Apellis Pharmaceuticals, a biopharmaceutical company focused on the development of novel therapeutic compounds to treat diseases through the inhibition of the complement system, announced positive results from the Phase 3 PEGASUS study evaluating pegcetacoplan (APL-2) in adults with PNH.
Top-line data show that pegcetacoplan met the study’s primary efficacy endpoint, demonstrating superiority to eculizumab with a significant improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16. The safety profile of pegcetacoplan was comparable with eculizumab in this study.
Click here to read the company’s full press release.
Read stories from patients within the Canadian PNH community.