Results 131 to 140 of about 7,776 (285)
Wearable sensors in paediatric neurology
Developmental Medicine &Child Neurology, Volume 67, Issue 7, Page 834-853, July 2025.Types of signals monitored in children's natural environments using wearable sensors, and their associated applications in various paediatric neurological conditions. Plain language summary: https://onlinelibrary.wiley.com/doi/10.1111/dmcn.16267 Abstract Wearable sensors have the potential to transform diagnosis, monitoring, and management of children ...
Camila González Barral, Laurent Servais
wiley +1 more source
Molecular Therapy: Methods & Clinical Development, 2021 This longitudinal cohort study aimed to determine whether circulating neurofilaments (NFs) can monitor response to molecular therapies in newborns with spinal muscular atrophy (SMA; NCT02831296).
Christiano R.R. Alves +13 more
doaj
Comprehensive Clinical Evaluation of Nusinersen Based on Multi-criteria Decision Analysis Method
Xiehe Yixue ZazhiObjectiveTo provide a theoretical basis for different drug decision-making scenarios by conducting a comprehensive clinical evaluation of nusinersen.MethodsBased on the method of multi-criteria decision analysis, a comprehensive clinical evaluation index
QU Jinghan +5 more
doaj +1 more source
Combinatorial treatment for spinal muscular atrophy [PDF]
, 2020 Spinal muscular atrophy (SMA) is a severe autosomal recessive motor neuron disease caused by loss of SMN1, which encodes a protein essential for motor neuron survival.
A. Poletti, K.H. Fischbeck
core +1 more source
Serious Neurologic Adverse Events in Tofersen Clinical Trials for Amyotrophic Lateral Sclerosis
Muscle &Nerve, Volume 71, Issue 6, Page 1006-1015, June 2025.ABSTRACT Introduction/Aims Tofersen is approved for the treatment of amyotrophic lateral sclerosis (ALS) due to superoxide dismutase 1 mutations (SOD1‐ALS). Here we report serious neurologic adverse events (AEs) that occurred in the tofersen clinical trials in people with SOD1‐ALS.
Alexandra Lovett +17 more
wiley +1 more source
Introduction to Novel Motor Neuron Disease [PDF]
, 2020 Motor neuron disease (MND) is a progressive and fatal neuromuscular disease; the most common and severe form of MND presentation is amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease.
de Fátima Ibañez Valdés, Lourdes +1 more
core +1 more source
Muscle &Nerve, Volume 71, Issue 6, Page 1016-1024, June 2025.ABSTRACT Introduction/Aims Survival Motor Neuron 1 (SMN1)‐related spinal muscular atrophy (SMA) is characterized by α‐motor neuron degeneration, with sensory function assumed to be clinically preserved. However, recent studies in severely affected patients and animal models have challenged this view.
Leandra A. A. Ros +7 more
wiley +1 more source
A multidisciplinary approach to dosing nusinersen for spinal muscular atrophy
Neurology Clinical Practice, 2019 In December 2016, nusinersen gained FDA approval as the first pharmacologic treatment for spinal muscular atrophy (SMA), a disorder of motor neurons and the leading genetic cause of infant mortality. Nusinersen's intrathecal delivery requirement, strict dosage protocol, and accelerated FDA approval presented a challenge to health care centers hoping to
Allan M. Glanzman +8 more
openaire +3 more sources
Frontiers in NeurologyBackground Spinal muscular atrophy (SMA) is a genetic progressive neuromuscular disease. Nusinersen is the first disease modifying drug approved to treat patients with SMA.
Yuyi Chen +8 more
semanticscholar +1 more source
Convective infux/glymphatic system: tracers injected into the CSF enter and leave the brain along separate periarterial basement membrane pathways [PDF]
, 2018 Tracers injected into CSF pass into the brain alongside arteries and out again. This has been recently termed the "glymphatic system" that proposes tracers enter the brain along periarterial "spaces" and leave the brain along the walls of veins.
Albargothy, Nazira J. +6 more
core +1 more source