We are excited to kick off February, the month of love, with our new #Love4Riaan Rare Disease Day Challenge! Our goal is to raise $28,000 in 28 days, in time to celebrate Rare Disease Day at the end of the month on February 28, 2022.
What is Rare Disease Day?
Rare Disease Day may mean different things to different people and to different rare disease communities. Ultimately though, it is a globally coordinated movement of human beings, from all over the world, impacted by a variety of rare diseases, coming together with purpose. To awaken others to the issues, to advocate for healthcare equity, to direct resources to the diagnosis, management, and treatment of rare diseases, which are in fact, not so rare. To share with the world our stories of struggle and resilience. Rare Disease Day is historically held at the end of February, i.e. the rarest day of the year.
To learn more about Rare Disease Day, please visit their website here.
How Can I Contribute to Rare Disease Day?
1.) Learn the facts about rare disease and spread the word this month, whether it’s via a contemplative social media post, or a casual conversation with a friend or co-worker! Rare diseases are not so rare: they impact over 300 million people worldwide. 50% of people impacted with rare disease are children, and 30% of these children will not live to see their 5th birthday. There are over 7,000 known rare diseases, most of them with a genetic cause. Over 95% of them lack approved therapies or treatments. Rare diseases impact people of all backgrounds, all over the world.
2.) Understand and spread the word that patient advocacy groups like ours are seeking to FUND a cure, especially as many of our diseases impact such small populations that there are no robust efforts to develop treatments by companies and institutions, with the urgency we require. The overarching issue is not that it is scientifically impossible to develop treatments: it’s the lack of funding. Indeed, science is advancing rapidly, it’s the funding that can’t keep up!
A lack of funding to develop well-known treatments like gene replacement therapy or to run drug repurposing screens therefore remains a substantial challenge for rare disease patient advocacy groups. All of these programs require significant resources, in the millions of dollars, as well as concerted advocacy from patient groups to facilitate data-sharing among scientists, and to keep programs moving at the speed and transparency our children require. We know where the cures and treatments are, we just need to fund them, together, as a community!
3.) Spread awareness about rare diseases and Cockayne Syndrome at your place of business, at school, or on social media in new and interesting ways! Email us at info@riaanresearch.org and we would be happy to share some resources and brainstorm ideas with you.
4.) Read and share the stories of families impacted by Cockayne Syndrome.
5.) Have influential friends in the media or elsewhere? Share this work with them, and see if they are interested in helping us amplify our message and this work, whether it’s via a retweet, or a news article.
6.) Hold a fundraiser of your own to raise money for Riaan Research Initiative! It can be a virtual auction, bakesale, a bike ride, a walk/run, an announcement at your place of worship, or just a peer-to-peer call to action via social media and email, like a Facebook fundraiser. We can help you set it up. Email us at info@riaanresearch.org.
7.) And of course, donate to our #Love4Riaan campaign, get your employer to match if possible, and spread the word! Any donation, of any size, will make a difference.
What will the #Love4Riaan ❤️ funds be used for?
Donations will be used to fund ongoing translational scientific research to develop treatments for Cockayne Syndrome which include: (1) drug repurposing (finding already FDA approved drugs that may halt or even reverse the neurodegeneration and other symptoms we see in CS (drug repurposing is the fastest path to a clinical trial), and the (2) CSA gene replacement therapy program (getting FDA investigational new drug approval on a gene therapy product to replace the mutated CSA gene with a healthy CSA gene and launch a clinical trial).
Riaan Research Initiative is the only 501(c)(3) patient advocacy organization exclusively devoted to finding treatments and therapies for Cockayne Syndrome.