Rafaelle’s story

My name is Rafaelle. I am Brazilian, I am 22 years old and I was diagnosed at age 5 with cystic fibrosis (CF) - F508 and S4X mutations. I have been on the lung transplant waiting list since February 2020.

My family sold everything to be able to buy 2 boxes (months) of Trikafta for me. After that, we found out about Gador through the CF Buyers Club and have fundraised in order to buy ten more boxes of Trixacar.

Thanks to this, I have recently celebrated 1 year of being on CF modulators. However, that money has also now run out and my family have been forced to take out a loan to buy more.

My story is just one of thousands of CF patients who cannot access the lifesaving drugs we need. Here is my reflection: What is the meaning of people, scientists, pharmacists, trained professionals devoting so much time, work, knowledge, research and technology to create something so incredible, capable of transforming people, giving back hope, dignity and saving lives, if the final product is inaccessible to those who need it?

What's the point of creating a formula that saves lives and choosing not to save them? How does Vertex feel to receive calls for help and ignore them, even though they have the miracle in their hands? I can't understand it.

We're not talking about cell phones, computers or a car - we're talking about lives. The power to end so much suffering, pain and sadness is in the hands of Vertex. A decision made by this company can save lives and change the history of cystic fibrosis across the world.