Castleman Disease Collaborative Network

Castleman Disease Collaborative Network

Patient groups like Beacon provide critical information and resources to help the rare disease community get closer to (repurposed) treatments for devastating illnesses. As someone who is literally alive today thanks to a repurposed drug that I discovered and tested...
Pitt Hopkins UK

Pitt Hopkins UK

I found out about Findacure (now Beacon) by chance back in 2014. There was going to be a workshop on Fundraising while I was in London, so I called Flóra to ask if I could come even though it was last moment. I didn’t realise that was one of the first workshops to be...
Wolfram Syndrome UK

Wolfram Syndrome UK

My name is Tracy Lynch and I’m the CEO and Co-Founder, alongside my husband, of Wolfram Syndrome UK (WSUK). I started my journey with the charity when we formed it 3 weeks after our daughter’s diagnosis in March 2010. There was no support for people and families...
FOP Friends

FOP Friends

I became a “rare leader” in 2009 when my eldest son was diagnosed with the ultra-rare genetic condition Fibrodysplasia Ossificans Progressiva (FOP). Since then, as a volunteer with zero experience, I’ve setup a patient organisation, worked with researchers, pharma,...
Meet Amanda, Trustee and Chair of The EOS Network

Meet Amanda, Trustee and Chair of The EOS Network

Amanda has double the inspiration for establishing The EOS Network. Amanda’s two children, Samuel and Heather, were born with eosinophilic gastrointestinal disease (EGID) which makes eating incredibly painful. When Amanda’s son was diagnosed with the disease at 7...
Meet Bhavna, CEO of Stargardt’s Connected

Meet Bhavna, CEO of Stargardt’s Connected

Bhavna’s world changed forever in September 2015 when her seven-year-old son, Ethan, was diagnosed with Stargardt’s: an inherited, degenerative retinal condition that slowly leads to sight loss. Ethan was struggling with his distance vision, so Bhavna went to the...
Metabolic Support UK

Metabolic Support UK

Metabolic Support UK (MSUK) are the leading organisation for Inherited Metabolic Disorders (IMDs), supporting thousands of patients worldwide through providing individual support, building patient communities, and continually advocating for and empowering those living...
Alexion, AstraZeneca Rare Disease

Alexion, AstraZeneca Rare Disease

A rare disease diagnosis can affect anyone at any time (the majority being children) and can have a significant impact on the quality of life of patients, their families, and caregivers. Alexion, AstraZeneca Rare Disease’s innovation is driven by understanding people...
Meet Wendy, Chair of the Norrie Disease Foundation

Meet Wendy, Chair of the Norrie Disease Foundation

When Wendy’s son was born with Norrie disease, a rare genetic condition that causes blindness, hearing loss and developmental delays, there was no UK organisation for her to turn to for support, and very little information available. Determined to support the Norrie...