Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study

James F. Howard, Kimiaki Utsugisawa, Michael Benatar, +614

The Lancet Neurology

Multicentre StudyPharmacologyMedicineMyasthenia GravisNeuromuscular Blockade +1

TDP-43 represses cryptic exon inclusion in the FTD–ALS gene UNC13A

X. Rosa, Mercedes Prudencio, Yuka Koike, +31

Nature

Establishment of In Vitro FUS-Associated Familial Amyotrophic Lateral Sclerosis Model Using Human Induced Pluripotent Stem Cells

Naoki Ichiyanagi, Koki Fujimori, Masato Yano, +16

Stem Cell Reports

Aberrant axon branching via Fos-B dysregulation in FUS-ALS motor neurons

Tetsuya Akiyama, Naoki Suzuki, Mitsuru Ishikawa, +28

EBioMedicine

Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension

Renato Mantegazza, Gil I. Wolfe, Srikanth Muppidi, +562

Neurology

FALS with FUS mutation in Japan, with early onset, rapid progress and basophilic inclusion

Naoki Suzuki, Masashi Aoki, Hitoshi Warita, +10

Journal of Human Genetics

BiologyMendelian DisorderEarly OnsetGenetic DisorderGenetics +7

Comprehensive targeted next-generation sequencing in Japanese familial amyotrophic lateral sclerosis

Ayumi Nishiyama, Tetsuya Niihori, Hitoshi Warita, +6

Neurobiology of Aging

Amyotrophic Lateral SclerosisMedicineGeneticsNext-generation SequencingNeurology +5

An Amyotrophic Lateral Sclerosis-Associated Mutant of C21ORF2 Is Stabilized by NEK1-Mediated Hyperphosphorylation and the Inability to Bind FBXO3

Yasuaki Watanabe, Tadashi Nakagawa, Tetsuya Akiyama, +5

iScience

Neuronal activity in the primate dorsomedial prefrontal cortex contributes to strategic selection of response tactics

Yoshiya Matsuzaka, Tetsuya Akiyama, Jun Tanji, +1

Proceedings of the National Academy of Sciences

TARDBP p.G376D mutation, found in rapid progressive familial ALS, induces mislocalization of TDP-43

Shio Mitsuzawa, Tetsuya Akiyama, Ayumi Nishiyama, +9

eNeurologicalSci

Unknown SignificanceEngineeringGeneticsGenetic EpidemiologyPathology +16