Daniela's Journey with Type 1 SMA

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

06/30/2025

About Daniela: Baby Daniela was diagnosed with Type 1 SMA via NIPT testing which was confirmed via amniocentesis.
She was born 4/14/2025 at 37 weeks old via C section after her plans for induction included staying with her mom for longer than we wanted.
Daniela was started on Evrysdi at 36 hours and has been continuing that treatment since.
Newborn screening confirmed (as we knew) she had Type 1 SMA and 2 copies of SMN 2.
She faced her first setback after we soon learned her antibodies of AAV9 were 1:200 and they needed to be at 1:50 or under to treat with a life changing gene therapy called zolgensma.
At 10.5 weeks we learned her levels came down allowing her to be treated with zolgensma on 7/9/25....
More to follow.
Be positive, thankful and remember to take a moment to look at not what you have, but who you have. Daniela has reaffirmed to us, family is everything.

06/30/2025

Daniela's 1st Chapter