Spinal muscular atrophy (SMA) type 1 is a genetic condition in which the nerves that control movement, breathing, and swallowing become progressively weaker and smaller. SMA has five types with ...

EXG001-307 is a recombinant AAV (rAAV) gene therapy in Phase 1/2 clinical development for Spinal Muscular Atrophy Type 1 in China. The company plans to file a US IND for EXG001-307 in 2024.

In July 2025, Novartis Pharmaceuticals conducted a Phase III multi-center, single dose (1.2 x 1014 vector genomes), ...

Baby Aasmika with SMA Type 1 received Zolgensma, the world’s most expensive gene therapy, thanks to India's largest-ever ...

SMA type 1. Type 1 is the most common form of the disease. According to existing data, an infant with this type has an average lifespan of 2 years or less.

Many children with type 1 SMA don’t live past 2 years old. Type 2. This type of SMA usually affects children starting between about 3 and 15 months old, before they can stand or walk by themselves.

Gök A, Saygılı SK, Kuruğoğlu S, Saltık S, Canpolat N. Children with type 1 spinal muscular atrophy are at increased risk for nephrolithiasis. Pediatr Neurol . 2023;150:32-36. doi:10.1016/j ...

Life expectancy for babies diagnosed with type 1 SMA is usually 2 years or less. Type 2. An intermediate form of the disease, type 2 usually appears when a child is between 7 and 18 months of age.

PSG was abnormal in 11 children (85%) with SMA type 1, 8 (42%) with SMA type 2, and 4 (31%) with SMA type 3. Despite the use of NIV by 11 children, none of the 47 children in the study met the ...

Key takeaways: At the time of last follow up, 82.6% of children born with SMA type 1 from 2016 on were still alive. Of the 140 type 1 SMA births between January 2010 and December 2015, 69% died as ...

He is Rafael, also known as Rafita, a 1-year-and-8-month-old baby from Ensenada who was diagnosed with spinal muscular atrophy type 1 when he was just one month old. Drake Bell Visits Baby in Ensenada ...