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Advances in the diagnosis of inherited neuromuscular diseases and implications for therapy development
Thompson R, Spendiff S, Roos A, et al.
The Lancet. Neurology, 2020, 19, 6, p 522
Nusinersen in adults with spinal muscular atrophy: new challenges
Mercuri E, Sansone V
The Lancet. Neurology, 2020, 19, 4, p 283
Leading the way for neuromuscular disorders since 1950
Collectif
The Lancet. Neurology, 2020, 19, 3, p 195
Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study
Hagenacker T, Wurster CD, Gunther R, et al.
The Lancet. Neurology, 2020, 19, 4, p 317
Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial
Hanna MG, Badrising UA, Benveniste O, et al.
The Lancet. Neurology, 2019, 18, 9, p 834
Long-term effect of thymectomy plus prednisone versus prednisone alone in patients with non-thymomatous myasthenia gravis: 2-year extension of the MGTX randomised trial
Wolfe GI, Kaminski HJ, Aban IB, et al.
The Lancet. Neurology, 2019, 18, 3, p 259
New therapies for neuromuscular diseases in 2018.
Shah AM, Al-Chalabi A
The Lancet. Neurology, 2019, 18, 1, p. 12-13
Endpoint choice for inclusion body myositis: a step too far?
Schmidt J
The Lancet. Neurology, 2019, 18, 9, p 807
Safety, tolerability, and preliminary efficacy of an IGF-1 mimetic in patients with spinal and bulbar muscular atrophy: a randomised, placebo-controlled trial.
Grunseich C, Miller R, Swan T, et al.
The Lancet. Neurology, 2018, 1043-1052
Classification and management of adult inflammatory myopathies
Selva-O'Callaghan A, Pinal-Fernández I, Trallero-Araguas E, et al.
The Lancet. Neurology, 2018, 17, 9, p 816
Cognitive behavioural therapy with optional graded exercise therapy in patients with severe fatigue with myotonic dystrophy type 1: a multicentre, single-blind, randomised trial
Okkersen K, Jimenez-Moreno C, Wenninger S, et al.
The Lancet. Neurology, 2018
Myotonic dystrophy type 1: reasons to be OPTIMISTIC
Gagnon C, Gallais B, Laberge L
The Lancet. Neurology, 2018
Neuromuscular disorders : 2017, a year to remember
Mercuri E
The Lancet. Neurology, 2018, 17, 1, p 12
IGF-1 for spinal and bulbar muscular atrophy: hope and challenges
Hashizume A, Hashizume A, Katsuno M
The Lancet. Neurology, 2018, 17, 12, p 1026
Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management
Birnkrant DJ, Bushby K, Bann CM, et al.
The Lancet. Neurology, 2018, 17, 3, p 251
Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management
Birnkrant DJ, Bushby K, Bann CM, et al.
The Lancet. Neurology, 2018, 17, 4, p 347
Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan
Birnkrant DJ, Bushby K, Bann CM, et al.
The Lancet. Neurology, 2018, 17, 5, p 445
Evidence-based care in Duchenne muscular dystrophy
McDonald CM, Mercuri E
The Lancet. Neurology, 2018, 17, 5, p 389
Genetic therapies for spinal muscular atrophy type 1
Aartsma-Rus A, Aartsma-Rus A
The Lancet. Neurology, 2017
Neuromuscular diseases: hope and hurdles in clinical trials
Verschuuren JJ, Verschuuren JJ
The Lancet. Neurology, 2017, 16, 1, p 12
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
Howard JF, Jr., Utsugisawa K, Benatar M, et al.
The Lancet. Neurology, 2017, 16, 12, p 976
Stakeholder collaboration for spinal muscular atrophy therapy development
Aartsma-Rus A, Balabanov P, Binetti L, et al.
The Lancet. Neurology, 2017, 16, 4, p 264
Another milestone in childhood spinal muscular atrophy
Wokke JH
The Lancet. Neurology, 2017, 16, 7, p 491
Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial
Bertini E, Dessaud E, Mercuri E, et al.
The Lancet. Neurology, 2017, 16, 7, p 513
Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial
Tardieu M, Zérah M, Gougeon ML, et al.
The Lancet. Neurology, 2017, 16, 9, p 712
