Canadian Association for Porphyria

Our Mission

The mission of the Canadian Association for Porphyria/Association Canadienne de Porphyrie is:
  • to deliver evidence-based information and support to patients with porphyria, their families, health care providers and the general public across Canada
  • to achieve standards and evidence-based comprehensive care for all people with porphyria throughout their lifespans.


Research for Canadians with Acute Hepatic Porphyria 

Deadline for Screening - June 29th, 2018

The Canadian Association for Porphyria/Association Canadienne de Porphyrie wants you to know about research that Alnylam is planning in Canada for patients with Acute Hepatic Porphyria. We have been limited in our ability to share this information as research sites are required to have all of the steps completed before they can begin to recruit participants. We are, however, concerned that a June 29th deadline for screening has recently been set. This might make it difficult for those who are interested in participating in the research to meet this deadline, so we have made the decision to share the information that we have.

Here is the link to the study.

ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)

We have been told by Alnylam that there are three research sites being set up in Canada: Edmonton, Montréal and Toronto. It is also possible for Canadians to participate in the United States studies if it is easier to travel to these sites (US sites are listed in the link). Arrangements can be made with Alnylam to cover travel costs.

The Edmonton location is the only Canadian research site that can release information about the Givosiran trials. Please contact Stephanie ASAP if you would like more information.


Edmonton, Alberta, Canada - Recruiting

Stephanie Nelson

Research Nurse, Clinical Trials Coordinator

Division of Hematology

Research Transition Facility

#4-016 8308 114 Street

Edmonton, Alberta Canada


(T) 780-492-3980

(F) 780-248-5899

If you are interested in finding out more about the research we encourage you to contact the nearest research site in Canada or the United States. We will forward information about the other research sites as it becomes available.

Summer Camp for Canadian children with Erythropoietic Protoporphyria

The Canadian Association for Porphyria/Associatio Porphyria/Association Canadienne de Porphyrie is pleased to announce that it has awarded travel grants to two very happy families to attend Camp Sundown, New York, USA.

It’s hard to imagine summer camp without sunshine, but for children with xeroderma pigmentosum (XP) or porphyria — two genetic conditions that cause severe injuries from UV rays — even the smallest amount of sunlight can be dangerous. At Camp Sundown in Craryville, children with these diseases can enjoy a week of swimming, arts and crafts, fishing, field trips, and most importantly, friendships with people just like them.   

Camp Sundown website, 2018

We would like to thank those who donated to this summer grant as they understand what attending this camp will mean to families. The CAP/ACP has committed to providing this grant again in 2019.

 Presentation is here!

CAP/ACP presented to the 

Canadian Blood Services Board Meeting 

December 7, 2017

The Canadian Association for Porphyria/Association Canadienne de Porphyrie (CAP) continues to advocate to improve care for Canadians with porphyria. Currently, patients with porphyria who need to access the only treatment available are having difficulty accessing it (Access to Heme Treatment in Canada 2016). As the treatment is a blood product we supported the application of Recordati in April, 2017 requesting that Canadian Blood Services (CBS) distribute this treatment. This request was refused in June, 2017 by the Provincial Territorial Blood Liaison Committee (PTBLC). 

The CAP/ACP has presented at the open board meeting of the CBS on June 22, 2017 and December 7, 2017.

Silvia Marchesin, on behalf of CAP presented our concerns and suggestions for a future collaborative process to the open board meeting of the CBS Board of Directors on June 22nd. Since this presentation, the proposal has been discussed by the PTBLC again in October and November but there has been no further decision. 

In order to continue advocating that CBS carry this blood product for Canadian patients with porphyria, Wendy Sauvé (President, CAP) presented to the CBS Open Board Meeting in Ottawa on December 7th, 2017. Her presentation is here: December 2017 Presentation to CBS Board.pptx (2).pdf

The 2017 Report Card on Canada's Blood System refers to how "short‐sighted financial considerations have prevented access to a number of blood and plasma‐derived therapies for certain rare conditions. For example: Normosang and Panhematin, red cell‐derived products to treat porphyria, are not distributed via CBS, making access uneven and often impossible. The product is routinely available in Quebec through Héma‐Québec." 

Canadian Hemophilia Society, 2017

The CAP/ACP Survey

Access to Heme Treatment in Canada 2016

Thank you to those who participated in the survey: Access to Heme Treatment in Canada. In order to better understand treatment for porphyria, the Canadian Association for Porphyria/Association Canadienne de Porphyrie (CAP/ACP) asked Canadians who have been diagnosed with acute porphyria  - Acute Intermittent Porphyria (AIP), Variegate Porphyria (VP), or Hereditary Coproporphyria (HCP) to take 5 - 10 minutes to complete a survey. We received 39 responses which clearly demonstrated that there is unequal access to Normosang and/or Panhematin treatment across Canada. 

For those diagnosed with an acute porphyria and told by a physician that they should receive Normosang and/or Panhematin only 50% (8 of 16 patients) were able to access treatment. Although some patients receive timely treatment, half of them suffer through attacks, receiving only supportive care (medication for nausea and pain) in Emergency or as an inpatient as they are not able to receive Normosang and/or Panhematin treatment.

The CAP/ACP asserts that patients with acute porphyria should have access to Normosang and/or Panhematin treatment, as needed, regardless of where they live in Canada. We are advocating for a future where these blood products will be distributed efficiently and equitably, and by so doing, greatly improve the lives of those suffering from acute hepatic porphyria.

Click here for a copy: Access to Heme Treatment in Canada 2016 

The International Congress on Porphyrins and Porphyrias (ICPP) 2017 was held in Bordeaux, France from June 25th to 28th, 2017. Three board members from the Canadian Association for Porphyria/Association Canadienne de Porphyrie attended the congress. We will be sharing new information from the congress soon.

How Can I Help?

"Patient self-organization is important for both mitigating uncertainty and driving progress toward improved treatments and outcomes for rare diseases."

Sara D. Khangura, et al., July 2015

Medical Disclaimer

Care has been taken to ensure that the information on this website is accurate at the time of publication. This information is, however, intended for general guidance only. The Canadian Association for Porphyria/Association Canadienne de Porphyrie disclaims for itself and for the author of this information, all responsibility for any mis-statements or for consequences of actions taken by any person while acting on information contained herein. Physicians and patients must make their own decisions according to the individual circumstances of each case.

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