Megan Willis' son, Edward, was given the gene therapy, Zolgensma, after being diagnosed with spinal muscular atrophy when he ...

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 ...

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

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 ...

Novartis’ ITVISMA, approved a month ago by the US FDA, used at Sheikh Khalifa Medical City under the supervision of the ...

"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 ...

Abu Dhabi marks landmark achievement in becoming the first in the world to deliver ITVISMA (onasemnogene abeparvovec) for the ...

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 ...

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.

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.