Medcare Royal Speciality Hospital (MRSH) in Dubai has become the world’s first hospital outside the USA, to offer a newly ...

After successfully raising the $2.4 million needed to save their child from a rare genetic disorder, baby Faziq finally ...

Forbes contributors publish independent expert analyses and insights. Spinal muscular atrophy affects the nerves that control muscle movement, leading to progressive weakening. As a result, infants ...

Novartis NVS announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) ...

Findings from a new study in the Journal of Neuromuscular Diseases, published by IOS Press, demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy ...

Cenrifki prevents MS disease progression and Itvisma delivers a healthy gene-to-cell nuclei, addressing the root cause of SMA.

Spinal muscular atrophy (SMA) is a rare, genetic condition that, across all types, affects ~1,600 people in the UK.1 It can cause muscle weakness which gets worse over time, causing movement, ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Credit: Novartis. The approval was supported by data from phase 3 STEER study, which enrolled patients with SMA who were treatment-naive and able to sit but never able to walk independently. Itvisma ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Topline results from a phase 3 clinical trial showed an ...

Regina Trollmann, MD, of the Division of Pediatric Neurology, Department of Pediatrics, Friedrich-Alexander-University of Erlangen-Nürnberg, Erlangen, Germany, and colleagues, did a retrospective ...

The AAV vector manufacturing market is mainly fueled by the expanding landscape of gene therapy research and development, as AAV vectors are extensively utilized for therapeutic gene delivery because ...