The FDA has approved Itvisma for the treatment of children two years and older, teens and adults living with spinal muscular ...

The 2025 Research Innovation Grants are supporting projects focused on biomarkers, risk factors, and treatments for ALS.

Parkinson's disease (PD) is an age-related, progressive neurodegenerative disorder. The hallmark of PD pathogenesis is the ...

Parkinson's disease (PD) is an age-related, progressive neurodegenerative disorder. The hallmark of PD pathogenesis is the ...

The FDA approved the intrathecal gene therapy onasemnogene abeparvovec (Itvisma) to treat spinal muscular atrophy (SMA) in ...

Itvisma contains the active ingredient in Zolgensma approved in 2019 for children younger than 2 years but is reformulated ...

Onasemnogene abeparvovec-brve is the first gene replacement therapy approved for SMA patients aged 2 years and older, expanding access beyond infants. Phase 3 trials demonstrated significant motor ...

Novartis wins FDA approval for Itvisma, a one-time gene therapy offering broader SMA treatment with sustained motor function ...

Researchers in Australia have identified a three-drug combination that greatly extends the survival of motor neuron cells in ...

ITVISMA is a prescription gene therapy used to treat adults and children 2 years of age and older with spinal muscular atrophy (SMA). ITVISMA is given as a one-time intrathecal injection. What is the ...

A 19-year-old who was diagnosed with a rare form of motor neurone disease (MND) in August has said it is "disgusting" that ...

Sinfield revealed this year's ultramarathon challenge - taking in more than 50km (31 miles) each day - would take place in ...