Lancet neurology
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Randomized Controlled Trial Multicenter Study
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial.
Risdiplam is an orally administered therapy that modifies pre-mRNA splicing of the survival of motor neuron 2 (SMN2) gene and is approved for the treatment of spinal muscular atrophy. The FIREFISH study is investigating the safety and efficacy of risdiplam in treated infants with type 1 spinal muscular atrophy versus historical controls. The primary endpoint of part 2 of the FIREFISH study showed that infants with type 1 spinal muscular atrophy attained the ability to sit without support for at least 5 s after 12 months of treatment. Here, we report on the safety and efficacy of risdiplam in FIREFISH part 2 over 24 months of treatment. ⋯ F Hoffmann-La Roche.
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A disconnect has developed over the past two decades between neurological and neuroscientific research, which have seen notable innovation and development, and our increasing understanding of the role of social and commercial determinants of health, including the health of the nervous system. Over the next two decades, grounding neurological research in public health and epidemiological principles can bring about a paradigm shift, away from reductionism, over-medicalisation, and health inequities towards neurological research that reduces inequalities and has true relevance to the populations it aims to serve. People who are involved in neurological and neuroscientific research and practice, as clinicians, researchers, publishers, and funders, can create change by being more aware of the social and commercial determinants of health, reprioritising research funding, and advocating for greater neurological health equity.