An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment ...

The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric ...

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an ...

Learn about the promising advance in prenatal treatment of spinal muscular atrophy, introducing a potential approach to suppress symptoms of the disorder in infants.

A toddler was successfully treated for a rare genetic disease, spinal muscular atrophy, after world-first in-womb therapy.

Spinal muscle atrophyorspinal muscular atrophy(SMA) is a genetic disorder that can affect babies, children, and adults. A ...

A two-and-a-half-year-old girl shows no signs of a rare genetic disorder, after becoming the first person to be treated for ...

Test your knowledge of spinal muscular atrophy (SMA) with this quiz covering its genetic causes, diagnosis, and treatment ...

SOUTH SAN FRANCISCO, CA, USA I 12, 2025 I Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved a New Drug ...

SMA is caused by a mutated or missing survival motor neuron 1 (SMN1) gene, leading to insufficient production of ... with ...

Infants with SMA type 1 born since 2016 experience better functional outcomes and life expectancy with disease-modifying treatments.

Spinraza treatment does not lead to significant kidney dysfunction in SMA patients with types 1 and 2, a safety and efficacy ...