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

The classification of spinal muscular atrophy (SMA) subtypes, the influence of SMN2 gene copies on disease severity, treatment effects on disease progression, age-related differences, long-term ...

Carbona struck a deal with Nationwide Children’s in late 2013, getting AveXis the exclusive right to develop an SMA treatment using the hospital’s inventions, including Kaspar’s, in exchange ...

Prenatal therapy for spinal muscular atrophy (SMA) with risdiplam has shown promise in a first-of-its-kind study led by Richard Finkel. Roche's Evrysdi has already shown profound benefits in ...

The classification of spinal muscular atrophy (SMA) subtypes, the influence of SMN2 gene copies on disease severity, treatment effects on disease progression, age-related differences, long-term ...

Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The first prenatal treatment for spinal muscular atrophy showed promise in a single case report. Risdiplam was given to ...

A study based on a survey of 50 adults linked poorer quality sleep with more depressive symptoms and respiratory issues in ...

SMA patients and advocacy groups have called for urgent action to make life-saving treatments more accessible. The cost of ...

The first prenatal therapy for spinal muscular atrophy (SMA) showed promising results, a case report indicated. More than 2 years after the child was born, no identifiable features of SMA have ...