The cerebellum, sensitive periods, and autism

SSH Wang, AD Kloth, A Badura - Neuron, 2014‏ - cell.com
Cerebellar research has focused principally on adult motor function. However, the
cerebellum also maintains abundant connections with nonmotor brain regions throughout …

Preventing amyotrophic lateral sclerosis: insights from pre-symptomatic neurodegenerative diseases

M Benatar, J Wuu, C McHutchison, RB Postuma… - Brain, 2022‏ - academic.oup.com
Significant progress has been made in understanding the pre-symptomatic phase of
amyotrophic lateral sclerosis. While much is still unknown, advances in other …

[HTML][HTML] Patient satisfaction with health care services; an application of physician's behavior as a moderator

F Manzoor, L Wei, A Hussain, M Asif… - International journal of …, 2019‏ - mdpi.com
Patient satisfaction is a measure of the extent to which a patient is content with the health
care they received from their health care provider. Patient satisfaction is one of the most …

[HTML][HTML] Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE …

C Darryl, E Bertini, KJ Swoboda, WL Hwu… - Neuromuscular …, 2019‏ - Elsevier
Spinal muscular atrophy (SMA) is a neurodegenerative disease associated with severe
muscle atrophy and weakness in the limbs and trunk. We report interim efficacy and safety …

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

R Masson, M Mazurkiewicz-Bełdzińska… - The Lancet …, 2022‏ - thelancet.com
Background 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 …

Malformations of cortical development: clinical features and genetic causes

R Guerrini, WB Dobyns - The Lancet Neurology, 2014‏ - thelancet.com
Malformations of cortical development are common causes of developmental delay and
epilepsy. Some patients have early, severe neurological impairment, but others have …

Genetic basis of brain malformations

E Parrini, V Conti, WB Dobyns, R Guerrini - Molecular syndromology, 2016‏ - karger.com
Malformations of cortical development (MCD) represent a major cause of developmental
disabilities, severe epilepsy, and reproductive disadvantage. Genes that have been …

Clinical trial and postmarketing safety of onasemnogene abeparvovec therapy

JW Day, JR Mendell, E Mercuri, RS Finkel, KA Strauss… - Drug safety, 2021‏ - Springer
Introduction This is the first description of safety data for intravenous onasemnogene
abeparvovec, the only approved systemically administered gene-replacement therapy for …

An integrated safety analysis of infants and children with symptomatic spinal muscular atrophy (SMA) treated with nusinersen in seven clinical trials

BT Darras, MA Farrar, E Mercuri, RS Finkel, R Foster… - CNS drugs, 2019‏ - Springer
Background Treatment with nusinersen has demonstrated significant and clinically
meaningful benefits in clinical trials in infants and children with spinal muscular atrophy …

[HTML][HTML] Epileptic multi-seizure type classification using electroencephalogram signals from the Temple University Hospital Seizure Corpus: A review

N McCallan, S Davidson, KY Ng, P Biglarbeigi… - Expert Systems with …, 2023‏ - Elsevier
Epilepsy is one of the most paramount neurological diseases, affecting about 1% of the
world's population. Seizure detection and classification are difficult tasks and are ongoing …