A 5-year-old boy who was born with a rare genetic condition is now able to walk by himself, his mother has said, after ...

A five-year-old boy who received the world's most expensive drug as a baby has made "incredible progress" and can walk ...

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 ...

Medcare Women & Children Hospital has successfully administered a pioneering intrathecal gene therapy for Spinal Muscular ...

Join the community workshop in Thiruvananthapuram to support families affected by Spinal Muscular Atrophy and learn about the condition.

A 5-year-old British boy with the rare disease spinal muscular atrophy (SMA) has miraculously learned to walk four years after receiving the world’s most expensive gene therapy. On the 31st, the BBC ...

A multidisciplinary panel of experts discusses the clinical burden of spinal muscular atrophy on patients, families, and caregivers and considers the importance of patient education and advocacy.

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 ...

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 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 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 ...