Rare Disease Day 2023- aHUS Community Project

On February 28, 2023, the world will join together to raise awareness for rare diseases like atypical HUS. Recognized annually on the last day of February, Rare Disease Day encourages patients and their families – as well as medical professionals, researchers, government officials, and companies – to join together and focus attention on rare diseases and how they impact people’s lives. Rare Disease Day began in 2008 as a EURORDIS initiative to raise rare disease awareness, and now it is truly a global event.

Last year adult aHUS patients and family caregivers of children joined in last year’s Rare Disease Day project to answer the prompt, “What would you like to know about aHUS?” There were 68 entries from 21 nations in 2022, which provided a unique look regarding what aHUS families want to know. Click HERE to view the final version of this aHUS project from last year’s Rare Disease Day. (Visit our Info Centre’s tab for Rare Disease Day & aHUS Awareness Day HERE, for projects and other resources.)

Our thanks to Jeff Schmidt, who has continued to lead these important insights into authentic snapshots of what it means to be living with atypical HUS. We’re pleased to announce details for the aHUS global community project for Rare Disease Day 2023.

2023 global aHUS community project for Rare Disease Day – by project lead Jeff Schmidt 

Rare Disease Day is under 2 months away!  On February 28, 2023, we will join together with our global aHUS family to raise awareness for rare diseases like aHUS. 

We all know the benefits of a complement inhibitor for treating aHUS compared with traditional ineffective treatments . For this year’s aHUS Alliance video project for Rare Disease Day, we would like to put the spotlight on side effects from infusions of complement inhibitors and any residual long term health events from having had aHUS.  From patients’ real life experience . This will help raise awareness of the aHUS community and help the newly formed aHUS CAB (community advisory board) in its efforts in dialogues with pharmaceutical companies about patient safety..

You may be thinking how can you participate since you have never received a complement inhibitor . We want to hear from you too as to why you have not been treated with a complement inhibitor. Maybe the doctor didn’t think you needed it, maybe you can’t get it in your country, or you can, but it is too expensive.  That information is very valuable to us too .  And if you have not had any side effects or long aHUS events that is important for us to know too.

Side effects have been defined as “An unexpected medical problem that happens during treatment with a drug or other therapy”. Such effects and events may be mild, moderate, or severe, and may be caused by something other than the drug or therapy being given.” These are sometimes called adverse events or AEs , the serious ones are SAEs.

By participating in this awareness project aHUS alliance Global Acftion might be contacted for more details. Pharma has an obligation to do so. If we tell you that there has been an interest in what you have reported you are under no obligation.  Regulations, rights, and responsibilities vary among nations.

How to participate in this important RDD 2023 project:

We are asking for those who want to participate in this year’s Rare Disease Day video, to send the following.

For those who have been treated with a complement inhibitor :

  – Your Photo

  – Name and where you are from

  

  • Answer “Yes”, “No”, or “I don’t know” for the following side effects /long term aHUS events listed ( see photo below too)

Side effect experiencedDue to aHUSDue to complement inhibitor
Headache  
Nausea  
Fatigue  
Dizziness  
Meningitis  
Other* ( specify if wish to)  

*specify any other side effect or long term aHUS event other than the above

Also answer answer “yes” or “no ” to the question “Do you know how to report a side effects”

For those who have not had a Complement Inhibitor :

Your photo

Name and where you are from

Answer “yes” ” no ” or “don’t know” to the following questions. ( see photo below)

Eculizumab is sold in my country

Ravulizumab is sold in my country

My Government funds treatment

Private insurance funds at least some of the cost

Jeff will then create a slide for you that will be posted on the aHUS Alliance page and the atypical HUS Families group.  And it will be included in the video/slideshow that will debut on Rare Disease Day.  In order to raise awareness leading up to Rare Disease Day we are hoping you will start sending in your submissions right away (don’t wait until the end) so we can post one or two a day from now until Rare Disease Day.

We welcome ALL those affected by aHUS from our global community to join in this project.

You can email your submission to Jeff Schmidt at jeff@ahusallianceaction.org

Submissions are due by the end of 2/17/2023 to allow time to put this together, but the earlier you can turn in your entry the better!

See samples below (thank you to Kerri Grey (son Ashley) and Kamal Shah for getting this started!).

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