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Comment on: The reliability of immunoassays to detect autoantibodies in patients with myositis is dependent on autoantibody specificity
Mahler M, Malyavantham K, Fritzler MJ, et al.
Rheumatology (Oxford, England), 2021, 60, 1, e35
Comment on: The reliability of immunoassays to detect autoantibodies in patients with myositis is dependent on autoantibody specificity
Sabour S
Rheumatology (Oxford, England), 2020
Myasthenic crises triggering Takotsubo cardiomyopathy
Scorza FA, Fiorini AC, Scorza CA, et al.
International journal of cardiology, 2020, 300, p 48
New developments in gene editing for Duchenne muscular dystrophy
Fernandez-Ruiz I
Nature Reviews. Cardiology, 2020
Resolution of pathogenic R-loops rescues motor neuron degeneration in spinal muscular atrophy
Hensel N, Detering NT, Walter LM, et al.
Brain : a journal of neurology, 2020, 143, 1, p 2
Novel candidate alleles associated with gene regulation for Emery-Dreifuss muscular dystrophy
Xiong H
EBiomedicine, 2020, 52
Comment on: "Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy" by Zhou et al
Chen TH
Journal of cachexia, sarcopenia and muscle, 2020
A decade of optimizing drug development for rare neuromuscular disorders through TACT
Wagner KR, de Luca A, Caizergues D, et al.
Nature reviews. Drug discovery, 2020, 19, 1, p 1
Hypoglycaemia in patients with type 1 SMA: an underdiagnosed problem?
Berti B, Onesimo R, Leone D, et al.
Archives of disease in childhood, 2019
Understanding factors hampering activities of daily living performance in childhood-onset myotonic dystrophy phenotypes
Gagnon C, Gallais B
Developmental medicine and child neurology, 2019
Spinal Muscular Atrophy Therapies: ICER Grounds the Price to Value Conversation in Facts
Starner CI, Gleason PP
Journal of managed care & specialty pharmacy, 2019, 25, 12, p 1306
Spinal Muscular Atrophy Diagnosed by Newborn Screening
Lopez-Chacon M, Buehner AN, Rao VK
Pediatric neurology briefs, 2019, 33, p 5
Une mutation du gène TNPO3 impliquée dans la LGMD D2 confère une protection à l’infection par le VIH-1 : The TNPO3 mutation that causes LGMD D2 induces protection against HIV-1 infection
Allamand V
Les Cahiers de Myologie, 2019, 35, HS2, p 45
Qualité de vie - Un prédicteur de mortalité dans l’insuffisance respiratoire sévère : Quality of life: a predictor of mortality in severe respiratory failure
Reveillere C
Les Cahiers de Myologie, 2019, 35, HS2, p 49
Score prédictif du risque de tachyarythmie ventriculaire dans les mutations du gène LMNA : Risk Prediction Score for Ventricular Tachyarrhythmias in LMNA mutations
Chapon F
Les Cahiers de Myologie, 2019, 35, HS2, p 50
La xénogreffe : un outil performant ! Protocole pour mieux suivre l’évolution d’une dystrophie musculaire : Xenograft: a powerful tool! Protocol to better follow the evolution of muscular dystrophy
Mornet D
Les Cahiers de Myologie, 2019, 35, HS2, p 47
MLASA1 is a poly-phenic but not a di-phenic condition
Finsterer J
Molecular genetics and metabolism reports, 2019, 21
Cladribine shows promise in patients with difficult-to-treat myasthenia gravis
Wood H
Nature reviews. Neurology, 2019
Reply: Treatment of anti-MDA5 autoantibody-positive juvenile dermatomyositis using tofacitinib
Landon-Cardinal O, Benveniste O, Allenbach Y
Brain : a journal of neurology, 2019
Treatment of anti-MDA5 autoantibody-positive juvenile dermatomyositis using tofacitinib
Sabbagh S, Almeida de Jesus A, Hwang S, et al.
Brain : a journal of neurology, 2019
Combined immunosuppression for ILD in myositis
Collison J
Nature Reviews Rheumatology (. nrrheum@nature.com.)
Nature reviews. Rheumatology, 2019
Commentary on "The Effect of Inspiratory Muscle Training on Duchenne Muscular Dystrophy: A Meta-analysis"
Moore JG, Cahalin LP
Pediatric physical therapy : the official publication of the Section on Pediatrics of the American Physical Therapy Association, 2019, 31, 4, p 330
Exercise training: Thinking ahead to counteract systemic manifestations of spinal muscular atrophy.
Alves CRR
The Journal of physiology, 2019
MGTX extension study longitudinally favors early thymectomy in non-thymomatous young-adult patients with AChR antibody-positive myasthenia gravis
Akaishi T, Motomura M, Aoki M, et al.
Annals of translational medicine, 2019, 7, s6
Better living through peptide-conjugated chemistry: next-generation antisense oligonucleotides
McNally EM, Leverson BD
The Journal of clinical investigation, 2019
