Hello and thank you
From a mom fighting for her daughter, your reading this letter and paying attention to our child’s story, even for a moment, means a tremendous amount to us.
When our daughter Kira was born, a tiny 5 lbs 4 ounces, eyes clouded by cataracts, she could only drink drops of milk off our fingertips. We were sent home from the hospital, alone and afraid, and over the next year slowly we made our own way to a diagnosis. It wasn’t easy, and I am eternally grateful for each person who took the time to help us along the way.
Kira has a disorder called Warburg Micro Syndrome (SPG69). It’s a type of progressing, complex spastic paraplegia that affects fewer than 200 people in the world.
These days our mission is simple — to find a drug that will slow the progression of Kira’s symptoms. She recently spent her third birthday doing her favorite thing — splashing around the pool in her father’s arms, giddy to feel the water rise up against her face. But the spasticity in her ankles is already getting worse, and we know that in five years time, her hands and her ankles will lock into contractures, her vision will go, and things like seizures, feeding issues, or breathing trouble might mean a happy day in the pool might just not be feasible.
Earlier this year, we created the Warburg Micro Research Foundation, because we wanted to make a difference in Kira’s life and the lives of others with the syndrome.
The time to act is now. Here’s why:
- In 2020, one of our board members, Dr. Mark Handley, made an important discovery suggesting Warburg Micro is a cholesterol biosynthesis disorder.
- In 2021 another of our board members began the Natural History Study for Early Onset HSPs, creating a repository for biological samples and paving the way for clinical trial readiness.
- In 2022 our scientific advisory board, including leading experts in motor neuron diseases, spastic paraplegias, and cholesterol disorders, met to discuss a roadmap to a treatment — the discrete next steps to FDA approval.
What we need is an animal model to test the theory that lipid modifying drugs can improve symptoms. And can you imagine if it works? Can you imagine if the only thing between my child and the painful progression of her symptoms is people who are paying attention? We have the team, we have the plan, and now, we just need your help.
Thank you from the bottom of our hearts,
The Warburg Micro Research Foundation