Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study

JF Howard, S Bresch, A Genge… - The Lancet …, 2023 - thelancet.com
Background Generalised myasthenia gravis is a chronic, unpredictable, and debilitating rare
disease, often accompanied by high treatment burden and with an unmet need for more …

Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 …

V Bril, A Drużdż, J Grosskreutz, AA Habib… - The Lancet …, 2023 - thelancet.com
Background Generalised myasthenia gravis is a chronic, unpredictable, and debilitating
autoimmune disease. New treatments for this disease are needed because conventional …

Guillain-Barré syndrome related to SARS-CoV-2 infection

K Bigaut, M Mallaret, S Baloglu, B Nemoz… - Neurology …, 2020 - AAN Enterprises
The outbreak of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2)
responsible for coronavirus disease 2019 (COVID-19) led to the death of thousands of …

[HTML][HTML] Anti-MOG antibodies are present in a subgroup of patients with a neuromyelitis optica phenotype

AK Pröbstel, G Rudolf, K Dornmair… - Journal of …, 2015 - Springer
Background Antibodies against myelin oligodendrocyte glycoprotein (MOG) have been
identified in a subgroup of pediatric patients with inflammatory demyelinating disease of the …

Natural History and Phenotypic Spectrum of GAA‐FGF14 Sporadic Late‐Onset Cerebellar Ataxia (SCA27B)

T Wirth, G Clément, C Delvallée, C Bonnet… - Movement …, 2023 - Wiley Online Library
Background Heterozygous GAA expansions in the FGF14 gene have been related to
autosomal dominant cerebellar ataxia (SCA27B‐MIM: 620174). Whether they represent a …

Impact of coronavirus disease 2019 in a French cohort of myasthenia gravis

G Solé, S Mathis, D Friedman, E Salort-Campana… - Neurology, 2021 - AAN Enterprises
Objective To describe the clinical characteristics and outcomes of coronavirus disease 2019
(COVID-19) among patients with myasthenia gravis (MG) and identify factors associated with …

Loss of phospholipase PLAAT3 causes a mixed lipodystrophic and neurological syndrome due to impaired PPARγ signaling

N Schuermans, S El Chehadeh, D Hemelsoet… - Nature …, 2023 - nature.com
Abstract Phospholipase A/acyltransferase 3 (PLAAT3) is a phospholipid-modifying enzyme
predominantly expressed in neural and white adipose tissue (WAT). It is a potential drug …

Adult-onset genetic leukoencephalopathies: a MRI pattern-based approach in a comprehensive study of 154 patients

X Ayrignac, C Carra-Dalliere, N Menjot de Champfleur… - Brain, 2015 - academic.oup.com
Inherited white matter diseases are rare and heterogeneous disorders usually encountered
in infancy. Adult-onset forms are increasingly recognized. Our objectives were to determine …

Biallelic RFC1-expansion in a French multicentric sporadic ataxia cohort

S Montaut, N Diedhiou, P Fahrer, C Marelli… - Journal of …, 2021 - Springer
Objective Cerebellar ataxia with neuropathy and vestibular areflexia syndrome (CANVAS) is
a recessively inherited multisystem ataxia compromising cerebellar, vestibular, and sensory …

Charcot–Marie–Tooth disease misdiagnosed as chronic inflammatory demyelinating polyradiculoneuropathy: An international multicentric retrospective study

F Hauw, G Fargeot, D Adams, S Attarian… - European journal of …, 2021 - Wiley Online Library
Abstract Background and purpose Charcot–Marie–Tooth (CMT) disease, an untreatable
hereditary polyneuropathy, may mimic chronic inflammatory demyelinating …