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.

Exegenesis Bio to Present 9-Patient Data from a Phase 1/2 Clinical Trial of EXG001-037, a Novel rAAV Gene Therapy for Spinal Muscular Atrophy (SMA) Type 1: Improved Head Control and Sitting ...

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

The life expectancy for a person with SMA depends on which type they have and new therapies that may extend their lifespan. Learn more.

According to researchers, the most prevalent risk factors in children with type 1 spinal muscular atrophy (SMA) are hypercalciuria and elevated urine specific gravity.

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

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.

Babies with type 1 might also struggle to get the nutrients they need because they have problems swallowing and sucking. Life expectancy for babies diagnosed with type 1 SMA is usually 2 years or ...

Disease-modifying therapies introduced in Italy led to better outcomes and survival rates in children born after 2016 with type 1 spinal muscular atrophy compared with those born prior to then ...

Spinal muscular atrophy is sorted into types, depending on the age and development milestones reached when symptoms first start.

Researchers evaluated oxygen saturation and arterial CO2 variables to screen for sleep disordered breathing in children with SMA type 1-3. Of the measures evaluated, only an oxygen desaturation ...

SMA Type 1 represents an estimated 50% of all cases, for which life expectancy is less than 18 months without treatment.