Half a million dollars!
July 2024
Dear Friends and Supporters of Conquering Gyrate Atrophy,
I hope you and your families are staying cool during this hot summer. We are excited to share some updates on the research we’ve supported. But first… please enroll in the GYROS study.
If you or a loved one has gyrate atrophy, please enroll in GYROS, the natural history study on GA. We need as many patients as possible to participate in this study that will lay the foundation for future clinical trials on GA. There are centers around the world and some travel funds are available. See here for more details and the list of centers currently certified. There are 5 more centers in process. If you have any trouble, please let us know. We are happy to connect you with the organizers to find a way for you to participate.
And now for the research. To date, we have committed over $500,000 towards GA research!
#1:
Dr. Katherine Uyhazi and colleagues from University of Pennsylvania recently shared some results at the 2024 ARVO conference. Using cells from people with GA, they found that the cells recovered when treated with gene therapy. This is BIG news. This is the first indication that gene therapy will work on GA! Without this, we could not move ahead. See here for the full abstract.
#2:
Drs. Jefferson Doyle, David Valle and Mandeep Singh are working to test gene therapy for GA targeting the liver. Since GA is a metabolic disease, there’s reason to think this may be an effective approach. A recent update from them shared very positive results. (Can’t share the details just yet)
#3:
We have recently awarded a grant to Dr. Nicola Brunetti-Pierri and colleagues at Telethon Institute of Genetics and Medicine in Italy (our first international award!) to study some safety issues with liver-directed gene therapy. For more details, see here.
#4:
GYROS, the natural history study is well underway with 11 participants already enrolled. The goal is to have 45 participants. Please help us spread the word!
And most importantly…
We began this work with a scientific symposium hosted by Johns Hopkins where a group of preeminent researchers came to discuss gyrate atrophy for the first time. Now we have researchers around the world working on gyrate atrophy and all of these researchers are communicating and collaborating with each other.
This is a direct result of patient advocates who are pushing researchers to talk to each other and with patients. We must shout out a special thanks to Patrick Schultink in the Netherlands and Graciela Chairez in Mexico who have both organized meetings with researchers on GA. It takes all of us to build momentum!
Of course, none of this would be possible without you. Each of you has helped to build awareness about this disease and support finding a cure. If we can get to a half million dollars in under 5 years, let’s get to a million in half that time. Please donate to continue this research.
We are just getting started and already so many people have helped us with every step. We are deeply grateful that you are on this journey with us.
Karabi and Malay Acharya
Founders, Conquering Gyrate Atrophy