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Alterations in Nodes of Ranvier and Schmidt-Lanterman Incisures in Charcot-Marie-Tooth Neuropathies.
- Published in:
- Annals of the New York Academy of Sciences, 1999, v. 883, n. 1, p. 508, doi. 10.1111/j.1749-6632.1999.tb08623.x
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- Publication type:
- Article
Peptide-Based Inhibition of NF- κRescues Diaphragm Muscle Contractile Dysfunction in a Murine Model of Duchenne Muscular Dystrophy.
- Published in:
- Molecular Medicine, 2011, v. 17, n. 5/6, p. 508, doi. 10.2119/molmed.2010.00263
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- Publication type:
- Article
Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy.
- Published in:
- Journal of Neuromuscular Diseases, 2024, v. 11, n. 3, p. 687, doi. 10.3233/JND-230185
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- Publication type:
- Article
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG).
- Published in:
- Journal of Neuromuscular Diseases, 2023, v. 10, n. 3, p. 389, doi. 10.3233/JND-221560
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- Publication type:
- Article
A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment.
- Published in:
- Journal of Neuromuscular Diseases, 2022, v. 9, n. 1, p. 39, doi. 10.3233/JND-210665
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- Publication type:
- Article
Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.
- Published in:
- Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 989, doi. 10.3233/JND-210643
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- Publication type:
- Article
Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls.
- Published in:
- Journal of Neuromuscular Diseases, 2021, v. 8, n. 4, p. 469, doi. 10.3233/JND-200548
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- Publication type:
- Article
Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States.
- Published in:
- Journal of Market Access & Health Policy, 2023, v. 11, n. 1, p. 1, doi. 10.1080/20016689.2023.2216518
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- Publication type:
- Article
Proof of Concept of the Ability of the Kinect to Quantify Upper Extremity Function in Dystrophinopathy.
- Published in:
- PLoS Currents, 2013, p. 566, doi. 10.1371/currents.md.9ab5d872bbb944c6035c9f9bfd314ee2
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- Publication type:
- Article
A Randomized, Double-Blind Trial of Lisinopril and Losartan for the Treatment of Cardiomyopathy in Duchenne Muscular Dystrophy.
- Published in:
- PLoS Currents, 2013, p. 72, doi. 10.1371/currents.md.2cc69a1dae4be7dfe2bcb420024ea865
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- Publication type:
- Article
Cyclosporine nephrotoxicity without major organ transplantation.
- Published in:
- 1987
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- Publication type:
- journal article
Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy.
- Published in:
- World Journal of Pediatrics, 2019, v. 15, n. 3, p. 219, doi. 10.1007/s12519-019-00242-6
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- Publication type:
- Article
Health-Related Quality of Life and Emotional Distress Among Mothers of Sons With Muscular Dystrophy as Compared to Sex- and Age Group–Matched Controls.
- Published in:
- Journal of Child Neurology, 2021, v. 36, n. 3, p. 177, doi. 10.1177/0883073820962927
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- Publication type:
- Article
Dexmedetomidine and ketamine sedation for muscle biopsies in patients with Duchenne muscular dystrophy.
- Published in:
- Pediatric Anesthesia, 2014, v. 24, n. 8, p. 851, doi. 10.1111/pan.12387
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- Publication type:
- Article
Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose-response Study in Mice and Nonhuman Primates.
- Published in:
- Molecular Therapy, 2015, v. 23, n. 3, p. 477, doi. 10.1038/mt.2014.210
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- Publication type:
- Article
A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy.
- Published in:
- Molecular Therapy, 2015, v. 23, n. 1, p. 192, doi. 10.1038/mt.2014.200
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- Publication type:
- Article
Charting a Clear Path: The ASGCT Standardized Pathways Conference.
- Published in:
- Molecular Therapy, 2014, v. 22, n. 7, p. 1235, doi. 10.1038/mt.2014.95
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- Publication type:
- Article
VIP-expressing Dendritic Cells Protect Against Spontaneous Autoimmune Peripheral Polyneuropathy.
- Published in:
- Molecular Therapy, 2014, v. 22, n. 7, p. 1353, doi. 10.1038/mt.2014.77
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- Publication type:
- Article
Vascular Delivery of rAAVrh74.MCK.GALGT2 to the Gastrocnemius Muscle of the Rhesus Macaque Stimulates the Expression of Dystrophin and Laminin α2 Surrogates.
- Published in:
- Molecular Therapy, 2014, v. 22, n. 4, p. 713, doi. 10.1038/mt.2013.246
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- Publication type:
- Article
AAV1.NT-3 Gene Therapy for Charcot-Marie-Tooth Neuropathy.
- Published in:
- Molecular Therapy, 2014, v. 22, n. 3, p. 511, doi. 10.1038/mt.2013.250
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- Publication type:
- Article
AAV-mediated Overexpression of Human α7 Integrin Leads to Histological and Functional Improvement in Dystrophic Mice.
- Published in:
- Molecular Therapy, 2013, v. 21, n. 3, p. 520, doi. 10.1038/mt.2012.281
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- Publication type:
- Article
Novel and recurrent EMD mutations in patients with Emery-Dreifuss muscular dystrophy, identify exon 2 as a mutation hot spot.
- Published in:
- Journal of Human Genetics, 2011, v. 56, n. 8, p. 589, doi. 10.1038/jhg.2011.65
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- Publication type:
- Article
Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model.
- Published in:
- Human Molecular Genetics, 2013, v. 22, n. 24, p. 4929, doi. 10.1093/hmg/ddt342
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- Publication type:
- Article
Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice.
- Published in:
- Nature Communications, 2017, v. 8, n. 1, p. 1, doi. 10.1038/s41467-017-01146-7
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- Publication type:
- Article
Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1‐Year Interim Results from Study SRP‐9001‐103 (ENDEAVOR).
- Published in:
- Annals of Neurology, 2023, v. 94, n. 5, p. 955, doi. 10.1002/ana.26755
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- Publication type:
- Article
Reply.
- Published in:
- 2017
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- Publication type:
- letter
Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.
- Published in:
- 2016
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- Publication type:
- journal article
Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.
- Published in:
- Annals of Neurology, 2015, v. 77, n. 4, p. 668, doi. 10.1002/ana.24365
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- Publication type:
- Article
Reply.
- Published in:
- Annals of Neurology, 2014, v. 75, n. 2, p. 329, doi. 10.1002/ana.24084
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- Publication type:
- Article
Eteplirsen for the treatment of Duchenne muscular dystrophy.
- Published in:
- Annals of Neurology, 2013, v. 74, n. 5, p. 637, doi. 10.1002/ana.23982
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- Publication type:
- Article
LTBP4 genotype predicts age of ambulatory loss in duchenne muscular dystrophy.
- Published in:
- Annals of Neurology, 2013, v. 73, n. 4, p. 481, doi. 10.1002/ana.23819
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- Publication type:
- Article
Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy.
- Published in:
- Pediatric Pulmonology, 2019, v. 54, n. 2, p. 179, doi. 10.1002/ppul.24203
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- Publication type:
- Article
AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models.
- Published in:
- Annals of Clinical & Translational Neurology, 2015, v. 2, n. 3, p. 256, doi. 10.1002/acn3.172
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- Publication type:
- Article
Validity of remote live stream video evaluation of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy.
- Published in:
- PLoS ONE, 2024, v. 19, n. 5, p. 1, doi. 10.1371/journal.pone.0300700
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- Publication type:
- Article
Study of mitochondrial DNA depletion in muscle by single-fiber polymerase chain reaction.
- Published in:
- 1998
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- Publication type:
- journal article
Amyloidosis causing a progressive myopathy.
- Published in:
- Muscle & Nerve, 1995, v. 18, n. 9, p. 1016, doi. 10.1002/mus.880180914
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- Publication type:
- Article
The dropped head syndrome with chronic inflammatory demyelinating polyneuropathy.
- Published in:
- Muscle & Nerve, 1994, v. 17, n. 7, p. 808, doi. 10.1002/mus.880170717
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- Publication type:
- Article
Adult acid maltase deficiency.
- Published in:
- Muscle & Nerve, 1993, v. 16, n. 6, p. 672, doi. 10.1002/mus.880160614
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- Publication type:
- Article
Case of the month. Latex hypersensitivity following exposure to gloves during electromyography.
- Published in:
- Muscle & Nerve, 1993, v. 16, n. 5, p. 559, doi. 10.1002/mus.880160520
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- Publication type:
- Article
Randomized, double-blind trial of mazindol in Duchenne dystrophy.
- Published in:
- Muscle & Nerve, 1990, v. 13, n. 12, p. 1169, doi. 10.1002/mus.880131212
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- Publication type:
- Article
Manual muscle testing.
- Published in:
- Muscle & Nerve, 1990, v. 13, n. S1, p. S16, doi. 10.1002/mus.880131307
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- Publication type:
- Article
Clinical investigation in Duchenne muscular dystrophy: Penicillamine and vitamin E.
- Published in:
- Muscle & Nerve, 1988, v. 11, n. 11, p. 1164, doi. 10.1002/mus.880111110
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- Publication type:
- Article
Studies on the pathogenesis of vincristine-induced neuropathy.
- Published in:
- Muscle & Nerve, 1987, v. 10, n. 1, p. 80, doi. 10.1002/mus.880100115
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- Publication type:
- Article
Clinical investigation in duchenne dystrophy. VI. Double-blind controlled trial of nifedipine.
- Published in:
- Muscle & Nerve, 1987, v. 10, n. 1, p. 22, doi. 10.1002/mus.880100106
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- Publication type:
- Article
Clinical investigation in Duchenne Dystrophy: V. Use of creatine kinase and pyruvate kinase in carrier detection.
- Published in:
- Muscle & Nerve, 1985, v. 8, n. 1, p. 60, doi. 10.1002/mus.880080111
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- Publication type:
- Article
Clinical investigation in duchenne muscular dystrophy: IV. Double. Blind controlled trial of leucine.
- Published in:
- Muscle & Nerve, 1984, v. 7, n. 7, p. 535, doi. 10.1002/mus.880070704
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- Publication type:
- Article
Clinical investigation in duchenne dystrophy: 2. Determination of the 'power' of therapeutic trials based on the natural history.
- Published in:
- Muscle & Nerve, 1983, v. 6, n. 2, p. 91, doi. 10.1002/mus.880060204
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- Publication type:
- Article
Clinical trial in duchenne dystrophy. I. The design of the protocol.
- Published in:
- Muscle & Nerve, 1981, v. 4, n. 3, p. 186, doi. 10.1002/mus.880040304
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- Publication type:
- Article
Lack of benefit of allopurinol in duchenne dystrophy.
- Published in:
- Muscle & Nerve, 1979, v. 2, n. 1, p. 53, doi. 10.1002/mus.880020108
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- Publication type:
- Article
Myoclonic epilepsy and ragged-red fibers with cytochrome oxidase deficiency: Neuropathology, biochemistry, and molecular genetics.
- Published in:
- Annals of Neurology, 1989, v. 26, n. 1, p. 20, doi. 10.1002/ana.410260104
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- Publication type:
- Article