About us

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.

The Association is generously supported by corporate and private donors, including unrestricted educational grants from Alexion Pharmaceuticals, Apellis Pharmaceuticals, and Ra Pharmaceuticals, Inc.

GET STARTED TODAY: Download for free and join the Anytime Access to PNH Health group!


New Phase 3 C3 inhibitor clinical trial begins

Apellis Pharmaceuticals Inc., a biopharmaceutical company focused on the development of novel therapeutic compounds to treat diseases through the inhibition of the complement system, has initiated its PEGASUS Phase 3 clinical trial assessing the safety and efficacy of APL-2, a novel inhibitor of complement factor C3, in patients with PNH compared to eculizumab. According to a company news release, PEGASUS is a 70-patient, randomized, head-to-head study comparing APL-2 monotherapy to eculizumab monotherapy in patients currently on treatment with eculizumab who have a hemoglobin level <10.5 g/dL, regardless of eculizumab dose or transfusion history.  APL-2 is being investigated with the goal of improving anemia and transfusion dependency in patients with PNH. Currently, the PEGASUS Phase 3 trial is recruiting PNH patients in the U.S., but the Canadian Association of PNH Patients has learned from Apellis that the company hopes to add other clinical trial centres in the near future, including in Canada. For more information on the PEGASUS Phase 3 clinical trial, visit ClinicalTrials.gov here.    
Read more

FIND A PNH SPECIALIST: View a list of board certified hematologists in your province