Nusinersen (Spinraza) and risdiplam (Evrysdi) are available on the NHS as possible treatments for spinal muscular atrophy (SMA). Nusinersen is a possible treatment for 5q SMA. Risdiplam is a possible ...

The National Institute for Health and Care Excellence (NICE) has recommended that two drugs for spinal muscular atrophy (SMA) — nusinersen (Spinraza, Biogen) and risdiplam (Evrysdi, Roche) — move to ...

Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers ...

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

Stop nusinersen if there is sustained deterioration across a range of clinical measures and the treatment is not providing benefit. Assess whether the person is benefitting from treatment based on ...

This new formulation provides an additional treatment option for pediatric and adult patients, making the therapy more accessible and convenient in tablet form. Evrysdi, marketed by Genentech, a Roche ...

The Muscular Dystrophy Association celebrates the FDA approval of Evrysdi in tablet form, a more accessible treatment option for people living with SMA. · GlobeNewswire Inc. This new formulation ...

Biogen has announced it will test its spinal muscular atrophy drug Spinraza in patients who still have unmet clinical needs after treatment with Novartis’ gene therapy Zolgensma. The planned RESPOND ...