Daniela's Journey with Type 1 SMA

06/03/2026

Daniela showing off her coordination! She is learning more each day! Marina MahoneyChris Mahoney

05/26/2026

Before 8 AM… and already crushing milestones 💪💜

This morning alone, Daniela walked with her walker for over 30 minutes total, waved to every dog in the neighborhood, and reminded us all what determination and joy look like. 🐶☀️

She’s growing stronger, happier, and more independent every single day — and we couldn’t be more proud of our sweet girl. Watching her take on the world with a smile is something we never take for granted.

One step at a time… and she keeps amazing us all. 💜

Marina Mahoney Chris Mahoney

05/10/2026

Just a little girl doing BIG things ❤️

Watching Daniela take steps with her walker this week was one of those moments we’ll never forget.

She refuses to crawl and is moving right along 🤣

SMA is not what it once was, and we hope sharing her journey gives other families hope for the future too.

Marina Mahoney Chris Mahoney

04/24/2026

Daniela is driving already! Chris Mahoney Marina Mahoney

04/01/2026

Daniela is thankful for Dr. Lee who always advocates for her and her health

Clinical trials bring cutting-edge treatments to patients. But what happens before clinical trials can begin? Natural history studies—and sometimes they save lives.

🔍 Natural history studies track the course of a disease over time, helping researchers understand how it develops, progresses, and affects daily life.

The neuromuscular research and data coordination center here at GCH played a key role in collecting this data for infants with Type 1 spinal muscular atrophy, which leads to muscle loss and impacts breathing, swallowing, walking and more. By closely tracking progression and survival, the team helped establish a clear picture of the disease without treatment. “We found there was only an 8% survival rate by 20 months,” explained Dr. Lee.

That starting point clearly demonstrated the urgency of new therapies, setting the stage for multiple clinical trials. 🧪

The result? Treatments showing the most drastic improvements, including gene therapy, were brought to patients faster and are now saving lives around the world. 👶🌎

03/24/2026