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Challenges associated with homologous directed repair using CRISPR-Cas9 and TALEN to edit the DMD genetic mutation in canine Duchenne muscular dystrophy
Mata Lopez S, Balog-Alvarez C, Vitha S, et al.
PLoS ONE, 2020, 15, 1
Omecamtiv mecarbil lowers the contractile deficit in a mouse model of nebulin-based nemaline myopathy
Lindqvist J, Lee EJ, Karimi E, et al.
PLoS ONE, 2019, 14, 11
Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy
Muntoni F, Domingos J, Manzur AY, et al.
PLoS ONE, 2019, 14, 9
Angiotensin II receptor blocker losartan exacerbates muscle damage and exhibits weak blood pressure-lowering activity in a dysferlin-null model of Limb-Girdle muscular dystrophy type 2B
White Z, Milad N, Tehrani AY, et al.
PLoS ONE, 2019, 14, 8
Investigating the clinical use of structured light plethysmography to assess lung function in children with neuromuscular disorders
Fleck D, Curry C, Donnan K, et al.
PLoS ONE, 2019, 14, 8
Discovery of potential urine-accessible metabolite biomarkers associated with muscle disease and corticosteroid response in the mdx mouse model for Duchenne
Thangarajh M, Zhang A, Gill K, et al.
PLoS ONE, 2019, 14, 7
Appendicular skeletal muscle mass: A more sensitive biomarker of disease severity than BMI in adults with mitochondrial diseases
Hou Y, Xie Z, Zhao X, et al.
PLoS ONE, 2019, 14, 7
The frequency of SMN gene variants lacking exon 7 and 8 is highly population dependent
Vijzelaar R, Snetselaar R, Clausen M, et al.
PLoS ONE, 2019, 14, 7
Correction: Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53
Brogna C, Coratt G, Pane M, et al.
PLoS ONE, 2019, 14, 7
Physical activity of children and adolescents with Charcot-Marie-Tooth neuropathies: A cross-sectional case-controlled study.
Kennedy RA, Carroll K, Paterson KL, et al.
PLoS ONE, 2019, 14, n6
Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53.
Brogna C, Coratti G, Pane M, et al.
PLoS ONE, 2019, 14, n6
Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents
Landrum Peay H, Fischer R, Tzeng JP, et al.
PLoS ONE, 2019, 14, n5, e0213649
Diaphragm sniff ultrasound: Normal values, relationship with sniff nasal pressure and accuracy for predicting respiratory involvement in patients with neuromuscular disorders
Fayssoil A, Nguyen LS, Ogna A, et al.
PLoS ONE, 2019, 14, 4
Effects of the selective inhibition of proteasome caspase-like activity by CLi a derivative of nor-cerpegin in dystrophic mdx mice
Hovhannisyan Y, Melikyan G, Mougenot N, et al.
PLoS ONE, 2019, 14, 4
Evaluation of intuitive trunk and non-intuitive leg sEMG control interfaces as command input for a 2-D Fitts's law style task
Verros S, Lucassen K, Hekman EEG, et al.
PLoS ONE, 2019, 14, 4
Trends in incidence, prevalence, and mortality of neuromuscular disease in Ontario, Canada: A population-based retrospective cohort study (2003-2014)
Rose L, McKim D, Leasa D, et al.
PLoS ONE, 2019, 14, 3
Aberrant insulin receptor expression is associated with insulin resistance and skeletal muscle atrophy in myotonic dystrophies
Renna LV, Bose F, Brigonzi E, et al.
PLoS ONE, 2019, 14, 3
Systemic nature of spinal muscular atrophy revealed by studying insurance claims
Lipnick SL, Agniel DM, Aggarwal R, et al.
PLoS ONE, 2019, 14, 3
CRISPR-cas gene-editing as plausible treatment of neuromuscular and nucleotide-repeat-expansion diseases: A systematic review
Babacic H, Mehta A, Merkel O, et al.
PLoS ONE, 2019, 14,2
Correction: Correction: Wild-Type Mouse Models to Screen Antisense Oligonucleotides for Exon-Skipping Efficacy in Duchenne Muscular Dystrophy
Collectif
PLoS ONE, 2019, 14, 2
Early short-term PXT3003 combinational therapy delays disease onset in a transgenic rat model of Charcot-Marie-Tooth disease 1A (CMT1A)
Prukop T, Prukop T, Stenzel J, et al.
PLoS ONE, 2019, 14, 1
Evaluation of potential effects of Plastin 3 overexpression and low-dose SMN-antisense oligonucleotides on putative biomarkers in spinal muscular atrophy mice
Strathmann EA, Strathmann EA, Peters M, et al.
PLoS ONE, 2018, 13, 9
Assessment of diaphragmatic thickness by ultrasonography in Duchenne muscular dystrophy (DMD) patients
Laviola M, Priori R, D'Angelo MG, et al.
PLoS ONE, 2018, 13, 7
Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study.
Chabanon A, Seferian AM, Daron A, et al.
PLoS ONE, 2018, e0201004
Ambulatory function in spinal muscular atrophy: Age-related patterns of progression
Montes J, McDermott MP, Mirek E, et al.
PLoS ONE, 2018, 13, 6
