Writing this letter feels surreal. When the girls were diagnosed, we had so much hope. We were going to treat them in a year! We would save them! We immersed ourselves in the research world and found a determination to persevere, even to succeed all thanks to your generous support and donations. But soon, the options on the horizon faded away and as time passed, we were forced to accept that every day our girls' bodies were becoming weaker than our resolve to fight. While they are stubborn and brave, 'Bad Batten' is taking its irreversible and heartbreaking toll on their little bodies. Our naive hopes of a miracle have evolved into an acceptance that science takes time. While waiting, we have done everything possible to give our girls the best quality of life and provide happiness. FINALLY, the science has emerged!

Ten months ago, we assembled a team of experts; rare disease leaders in research, drug manufacturing, drug safety, regulatory and clinical trials. This team has been diligently and methodically working towards crafting an N of 2 clinical trial specific to Amelia and Makenzie; providing them one true chance at improving their quality of life. We have confidence in the researchers and scientific advisors that have been on this journey with us since diagnosis. What separates us from treating our girls is $1.2M out the $5.4M budget for Project Butterfly. Once we are able to secure this additional funding, treatment will begin as soon as May 2024!

We hope our story is part of the greater picture of personalized medicine as well as paving the way for future diagnosed children to gain access to treatment earlier. While we wish we could be treating the girls of seven years ago, today is still better than tomorrow. Every day matters in preserving their quality of life, and time is no longer on our side. The fragility of our daughters' lives cannot be over emphasized, and we need your help. Years ago when Amelia could better verbalize, she voiced her physical pain, her fear and asked doctors to make her better. We miss Amelia's voice. We do not want to lose Makenzie's voice too. Yet it is happening. Makenzie says it best...

"Imanptntptnt"

Translated by mom: "I am an impatient patient"

Makenzie Kahn, Age 13

With Hope,

Karen & David

Project Butterfly

An N-of-2 Trial treating a unique mutation of CLN3 and creating a road map for the future of personalized medicine.