Found: 54
Select item for more details and to access through your institution.
Transmembrane electrical characteristics of cultured human skeletal muscle cells.
- Published in:
- Journal of Cellular Physiology, 1987, v. 133, n. 2, p. 409, doi. 10.1002/jcp.1041330230
- By:
- Publication type:
- Article
Social Media and Clinical Trials in Pediatric Disease: Musings From the Bench.
- Published in:
- Frontiers in Genetics, 2021, v. 12, p. 1, doi. 10.3389/fgene.2021.734691
- By:
- Publication type:
- Article
Vitamin E Deficiency Neuropathy in Children with Fat Malabsorption Studies in Cystic Fibrosis and Chronic Cholestasis<sup>a</sup>.
- Published in:
- Annals of the New York Academy of Sciences, 1989, v. 570, n. 1, p. 156, doi. 10.1111/j.1749-6632.1989.tb14916.x
- By:
- Publication type:
- Article
Mutations in the skeletal muscle α-actin gene in patients with actin myopathy and nemaline myopathy.
- Published in:
- Nature Genetics, 1999, v. 23, n. 2, p. 208, doi. 10.1038/13837
- By:
- Publication type:
- Article
Pediatric Aspects of Spinal Rehabilitation.
- Published in:
- Neurorehabilitation & Neural Repair, 1994, v. 8, n. 1, p. 41, doi. 10.1177/136140969400800107
- By:
- Publication type:
- Article
Combination molecular therapies for spinal muscular atrophy: How much is enough?
- Published in:
- Muscle & Nerve, 2021, v. 63, n. 3, p. 279, doi. 10.1002/mus.27109
- By:
- Publication type:
- Article
Response to treatment in pediatric ocular myasthenia gravis.
- Published in:
- 2020
- By:
- Publication type:
- journal article
Twice‐weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy.
- Published in:
- Muscle & Nerve, 2019, v. 59, n. 6, p. 650, doi. 10.1002/mus.26441
- By:
- Publication type:
- Article
Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy.
- Published in:
- 2019
- By:
- Publication type:
- journal article
Rasch analysis of clinical outcome measures in spinal muscular atrophy.
- Published in:
- Muscle & Nerve, 2014, v. 49, n. 3, p. 422, doi. 10.1002/mus.23937
- By:
- Publication type:
- Article
Liquid formulation of pentoxifylline is a poorly tolerated treatment for duchenne dystrophy.
- Published in:
- Muscle & Nerve, 2011, v. 44, n. 2, p. 170, doi. 10.1002/mus.22127
- By:
- Publication type:
- Article
Restoration of Nusinersen Levels Following Treatment Interruption in People With Spinal Muscular Atrophy: Simulations Based on a Population Pharmacokinetic Model.
- Published in:
- CNS Drugs, 2022, v. 36, n. 2, p. 181, doi. 10.1007/s40263-022-00899-0
- By:
- Publication type:
- Article
Medical management of muscle weakness in Duchenne muscular dystrophy.
- Published in:
- PLoS ONE, 2020, v. 15, n. 10, p. 1, doi. 10.1371/journal.pone.0240687
- By:
- 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
- By:
- Publication type:
- Article
CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy.
- Published in:
- Annals of Neurology, 2005, v. 58, n. 1, p. 151
- By:
- Publication type:
- Article
CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy.
- Published in:
- Annals of Neurology, 2005, v. 58, n. 1, p. 151
- By:
- Publication type:
- Article
Heterogeneity of nemaline myopathy cases with skeletal muscle α‐actin gene mutationsSee Appendix on page 93.
- Published in:
- Annals of Neurology, 2004, v. 56, n. 1, p. 86
- By:
- Publication type:
- Article
Nemaline myopathy: a clinical study of 143 cases.
- Published in:
- 2001
- By:
- Publication type:
- journal article
Respiratory Care of the Patient with Duchenne Muscular Dystrophy.
- Published in:
- American Journal of Respiratory & Critical Care Medicine, 2004, v. 170, n. 4, p. 456, doi. 10.1164/rccm.200307-885ST
- By:
- Publication type:
- Article
Immune-mediated myositis following gene therapy for Duchenne muscular dystrophy: a case report.
- Published in:
- Journal of Neurology, 2024, v. 271, n. 8, p. 5659, doi. 10.1007/s00415-024-12431-z
- By:
- Publication type:
- Article
Estimating health state utilities in Duchenne muscular dystrophy using the health utilities index and EQ-5D-5L.
- Published in:
- Journal of Patient-Reported Outcomes, 2023, v. 7, n. 1, p. 1, doi. 10.1186/s41687-023-00671-y
- By:
- Publication type:
- Article
Dietary L-Tyrosine Supplementation in Nemaline Myopathy.
- Published in:
- Journal of Child Neurology, 2008, v. 23, n. 6, p. 609, doi. 10.1177/0883073807309794
- By:
- Publication type:
- Article
Congenital Bone Fractures in Spinal Muscular Atrophy: Functional Role for SMN Protein in Bone Remodeling.
- Published in:
- Journal of Child Neurology, 2007, v. 22, n. 8, p. 967, doi. 10.1177/0883073807305664
- By:
- Publication type:
- Article
27TH ANNUAL CARRELL-KRUSEN SYMPOSIUM.
- Published in:
- 2005
- By:
- Publication type:
- Proceeding
Meeting Abstracts.
- Published in:
- Journal of Child Neurology, 2004, v. 19, n. 3, p. 201
- By:
- Publication type:
- Article
Meeting Abstracts.
- Published in:
- Journal of Child Neurology, 2003, v. 18, n. 3, p. 203
- By:
- Publication type:
- Article
Postoperative Malnutrition in Duchenne Muscular Dystrophy.
- Published in:
- Journal of Child Neurology, 2003, v. 18, n. 1, p. 17, doi. 10.1177/08830738030180011201
- By:
- Publication type:
- Article
Meeting Abstracts.
- Published in:
- Journal of Child Neurology, 2002, v. 17, n. 3, p. 206
- By:
- Publication type:
- Article
Assessment of Cardiac Function in Adolescents With Duchenne Muscular Dystrophy: Importance of Neurohormones.
- Published in:
- Journal of Child Neurology, 2002, v. 17, n. 3, p. 191, doi. 10.1177/088307380201700307
- By:
- Publication type:
- Article
Meeting Abstracts.
- Published in:
- Journal of Child Neurology, 2001, v. 16, n. 3, p. 203, doi. 10.1177/088307380101600308
- By:
- Publication type:
- Article
Abstracts.
- Published in:
- 1999
- By:
- Publication type:
- Abstract
Muscle Fatigue in Spinal Muscular Atrophy.
- Published in:
- Journal of Child Neurology, 1997, v. 12, n. 5, p. 321
- By:
- Publication type:
- Article
Carbamazepine Trial for Lesch-Nyhan Self-Mutilation.
- Published in:
- Journal of Child Neurology, 1996, v. 11, n. 6, p. 476, doi. 10.1177/088307389601100613
- By:
- Publication type:
- Article
Book Review: Neurological Rehabilitation, 2nd edition, edited by L. S. Illis. Published in 1994 by Blackwell Scientific, Oxford, England, 592 pages, $125.00.
- Published in:
- Journal of Child Neurology, 1996, v. 11, n. 4, p. 350, doi. 10.1177/088307389601100423
- By:
- Publication type:
- Article
Expert Witness Testimony.
- Published in:
- Journal of Child Neurology, 1996, v. 11, n. 2, p. 171, doi. 10.1177/088307389601100225
- By:
- Publication type:
- Article
Dystrophin and DNA Diagnosis in a Large Pediatric Muscle Clinic.
- Published in:
- Journal of Child Neurology, 1994, v. 9, n. 2, p. 162, doi. 10.1177/088307389400900211
- By:
- Publication type:
- Article
Book Review: Dystrophia Myotonica and Related Disorders, 2nd ed, revised by J.E. Caughey, Constantina Vayenas, and Joanne Dixon. Published in 1991 by C/-Pre-Print Productions, Auckland, New Zealand, 230 pages, $27.79 (paperback).
- Published in:
- Journal of Child Neurology, 1993, v. 8, n. 2, p. 195, doi. 10.1177/088307389300800226
- By:
- Publication type:
- Article
Book Review: Peripheral Neuropathy in Childhood, The International Review of Child Neurology, by R.A. Ouvrier, J.G. McLeod, and J.D. Pollard. Published in 1990 by Raven Press, New York, 252 pages, $84.00.
- Published in:
- Journal of Child Neurology, 1992, v. 7, n. 3, p. 322, doi. 10.1177/088307389200700318
- By:
- Publication type:
- Article
Myogenes and Myotubes.
- Published in:
- Journal of Child Neurology, 1992, v. 7, n. 2, p. 180, doi. 10.1177/088307389200700208
- By:
- Publication type:
- Article
Medullary Nuclear Dysplasia or Necrosis.
- Published in:
- Journal of Child Neurology, 1990, v. 5, n. 3, p. 260, doi. 10.1177/088307389000500324
- By:
- Publication type:
- Article
Long-term Mechanical Ventilation In Infants With Neuromuscular Disease.
- Published in:
- Journal of Child Neurology, 1988, v. 3, n. 1, p. 30, doi. 10.1177/088307388800300106
- By:
- Publication type:
- Article
Decreased Creatine Kinase Activity in Cultured Duchenne Dystrophic Muscle Cells.
- Published in:
- Journal of Child Neurology, 1987, v. 2, n. 1, p. 17, doi. 10.1177/088307388700200103
- By:
- Publication type:
- Article
Novel LMNA mutations in patients with Emery-Dreifuss muscular dystrophy and functional characterization of four LMNA mutations.
- Published in:
- Human Mutation, 2011, v. 32, n. 2, p. 152, doi. 10.1002/humu.21361
- By:
- Publication type:
- Article
Observing the Clinical Course of Duchenne Muscular Dystrophy in Medicaid Real-World Healthcare Data.
- Published in:
- Advances in Therapy, 2024, v. 41, n. 6, p. 2519, doi. 10.1007/s12325-024-02865-2
- By:
- Publication type:
- Article
Adherence and Persistence Among Risdiplam-Treated Individuals with Spinal Muscular Atrophy: A Retrospective Claims Analysis.
- Published in:
- Advances in Therapy, 2024, v. 41, n. 6, p. 2446, doi. 10.1007/s12325-024-02850-9
- By:
- Publication type:
- Article
Adherence and Persistence to Nusinersen for Spinal Muscular Atrophy: A US Claims-Based Analysis.
- Published in:
- Advances in Therapy, 2023, v. 40, n. 3, p. 903, doi. 10.1007/s12325-022-02376-y
- By:
- Publication type:
- Article
Normal vitamin E status in spinal muscular atrophy.
- Published in:
- Annals of Neurology, 1983, v. 13, n. 3, p. 328, doi. 10.1002/ana.410130318
- By:
- Publication type:
- Article
Cylindrical spirals in a familial neuromuscular disorder.
- Published in:
- Annals of Neurology, 1980, v. 7, n. 6, p. 550, doi. 10.1002/ana.410070608
- By:
- Publication type:
- Article
Baseline results of the Neuro NEXT spinal muscular atrophy infant biomarker study.
- Published in:
- Annals of Clinical & Translational Neurology, 2016, v. 3, n. 2, p. 132, doi. 10.1002/acn3.283
- By:
- Publication type:
- Article
Cooperative study for the assessment of therapeutic trials for the spinal muscular atrophies. Preliminary observations on the reliability of the DCN-SMA study group methodology.
- Published in:
- Muscle & Nerve, 1990, v. 13, n. S1, p. S11, doi. 10.1002/mus.880131305
- By:
- Publication type:
- Article