The cost burden of patients with SMA is considerable, and is estimated to be approximately $4 million to $5 million over 10 years in patients with early-onset SMA. This cost is 54.2 times greater than ...

The effects allowed these minors who could sit but not stand to move like they've never done before, including walking and ...

In an exclusive interaction with Professor Philip J Young from the University of Warwick we understood the importance of ...

"The availability of disease-modifying therapies for patients with other types of SMA suggests that these key disease characteristics in SMA 3 patients should be amenable to therapy." — Barry J. Byrne ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

Spinal muscular atrophy (SMA) is a genetic disorder caused by a homozygous recessive mutation in a gene on chromosome 5 (5q13), called the survival motor neuron 1 (SMN1) gene. It is due to deletion ...

The FDA has approved an adeno-associated virus 9 gene replacement therapy to treat individuals aged 2 years and older with ...

A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would ...

The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal ...

Spinal muscular atrophy (SMA) is a genetic condition in which the anterior horn motor neurons, as well as other lower motor neurons in the brain stem nuclei, degenerate due to mutations in the ...

Neurology biotech Voyager Therapeutics has laid off 30 employees, a spokesperson for the Massachusetts-based company ...

India makes many of the world’s drugs, but treatments for rare diseases like spinal muscular atrophy are imported and prohibitively costly. In desperation, parents are raising funds on social media.