Motor Function
Tofersen improves functional independence in SOD-1 ALS
Treatment with tofersen in patients with superoxide 1 dismutase 1 ALS was linked to preservation of motor function, maintenance of muscle strength and reduction of disease-specific biomarkers, data show.
Apitegromab improves motor function in spinal muscular atrophy as early as week 8
Topline results from the phase 3 SAPPHIRE clinical trial showed that an investigational muscle-targeted therapy improved motor function in patients with spinal muscular atrophy compared with placebo as early as week 8.
Higher dose of Spinraza improves motor function in infants with spinal muscular atrophy
Topline data from an ongoing clinical trial of a higher dose regimen of Spinraza in treatment-naive, symptomatic infants with spinal muscular atrophy showed significantly improved motor function at 6 months.
Spinal muscular atrophy therapy leads to sustained motor function improvement
An investigational, fully human monoclonal antibody developed as a muscle-targeted therapy for spinal muscular atrophy showed sustained motor function improvements at 48 months, according to the manufacturer.
Interim phase 1/2a study results positive for investigational Parkinson’s cell therapy
A South Korean biomedical company has announced positive 1-year assessment data from a phase 1/2a clinical trial of a human embryonic stem cell-derived midbrain dopaminergic progenitor for Parkinson’s disease.
Evrysdi linked to significantly improved outcomes in spinal muscular atrophy at 5 years
Newly released data from the open-label extension of the FIREFISH study confirmed sustained safety and efficacy of Evrysdi in children with type 1 spinal muscular atrophy over 5 years.
Nusinersen linked to improved motor function in spinal muscular atrophy
Treatment with nusinersen was linked to increased motor function and reduction of the neurodegenerative biomarker neurofilament light chain in children with spinal muscular atrophy, according to interim results of a study.
Apitegromab linked to improved outcomes for those with spinal muscular atrophy
Treatment with apitegromab was linked to improved muscle strength and range of motion, while being safe and well-tolerated in those with spinal muscular atrophy types 2 and 3, according to a poster presentation.
Givinostat improves range of motion, reduces decline of muscle function in DMD
Treatment with givinostat for ambulatory boys with Duchenne muscular dystrophy led to improved motion and reduced decline of muscle function and strength at 18 months, according to a poster at the 2024 MDA Clinical & Scientific Conference.
Zolgensma improves motor function at 52 weeks in spinal muscular atrophy
Treatment with Zolgensma improved motor function in a cohort of children with spinal muscular atrophy, according to a poster at the 2024 MDA Clinical & Scientific Conference.