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

The first prenatal therapy for spinal muscular atrophy (SMA) showed promising results, a case report indicated. More than 2 ...

Researchers at the University of Pittsburgh say a new minimally invasive intervention can target the root cause of neural function loss.

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude ...

Spinal muscular atrophy ... of motor neuron degeneration that usually starts during foetal development and is one of the most common genetic causes of infant death. SMA is caused by autosomal ...

1 Due to the degenerative nature of the disease, people with SMA will experience a decline in muscle strength throughout ... sense of urgency to address motor neuron loss in SMA from the first ...

SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein ... leading to muscle weakness over time. Depending on the type of SMA, an ...

Spinal muscular atrophy (SMA ... irreversible loss of motor neurons. SMA affects approximately one in 10,000 live births worldwide. The loss of motor neurons affects muscle functions, like ...

pre-mRNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival of motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home or on ...