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

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

A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers ...

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

The FDA approved Itvisma gene therapy for patients aged 2 years and older with spinal muscular atrophy and confirmed SMN1 ...

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

Opportunities in the SMA market include leveraging the launch of new myostatin inhibitors, expanding Zolgensma formulations to increase patient eligibility, and capitalizing on cheap nusinersen ...

Novartis’ Itvisma (onasemnogene) has been approved by the US FDA to treat spinal muscular atrophy (SMA) in children aged two years and older, teenagers and adults with a confirmed mutation in the ...

Emily Rogers, 6, is in hospital at Starship in Auckland rather than at home for Christmas. This photo of her is among those ...

A group of residents in Indore has launched a campaign to raise Rs 9 crore to save the life of a three-year-old girl suffering from a rare genetic disorder called Spinal Muscular Atrophy (SMA) Type 2.

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

A new review of studies reveals that incorporating SMA into newborn screening programs yields clinical improvements and ...