Help find a cure for SMA-PME & Farber Disease

Help Us Fund a Cure for Children Like Karina and Zayd

Karina and Zayd are two beautiful, brave children living with devastating, ultra-rare genetic diseases caused by ASAH1 gene mutations — leading to SMA-PME and Farber Disease. These conditions slowly rob children of their ability to move, eat, speak, and breathe comfortably. SMA-PME and Farber Disease are progressive, painful, life-limiting, and currently have no cure.

We are working to change that, and we need your help!

Karina, born in 2021, was a joyful little girl who loved unicorns and ballet dancing. But at age 3, her family noticed something was wrong — she was struggling to stand. Within months, she lost the ability to walk, then began experiencing seizures. After a long and painful months-long search for answers, she was diagnosed with SMA-PME. Today, Karina can no longer walk and suffers daily from epilepsy, but her spirit remains full of love and joy. Her family holds out hope for a treatment for Karina.

Zayd is a resilient, loving baby boy with the brightest smile who loves music, books, and going to the park. He was born in 2024 with a soft cry and subtle signs that something wasn’t right. By 8 weeks old, he was struggling with head control, muscle stiffness, and severe joint pain when picked up or moved. Following multiple specialist appointments and various tests, Zayd was 5 months old when he was diagnosed with Farber Disease type 1, a severe and devastating form of ASAH1-related disease that causes joint inflammation, hypotonia, a progressively hoarse voice, developmental delays, and difficulty gaining weight. Zayd is in physical & feeding therapy and fighting hard to grow stronger — but time is critical.

This fundraiser is created by Karina and Zayd’s parents, united by hope, love, and an urgent mission: to help fund the research that could save their children's lives — and the lives of others like them.

Research on gene therapy is underway, but funding is desperately needed to accelerate treatment development. Your donation can help give kids like Karina and Zayd a fighting chance at a future full of joy, movement, and life.

Karina and Zayd are only two of the children affected by these devastating conditions. Visit https://sma-pme.org/ to learn more about the stories of Adeline, Brayden, Kyndall, Noah, and others.

Let’s fight for a cure. Let’s fight for their childhoods.

#EndSMA_PME #FightFarber