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 ...

The newly licensed intrathecal gene therapy expands life-changing treatment options for children and adults affected by ...

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

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 ...

"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 ...

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 ...

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.

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 ...

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.