Works matching DE "TREATMENT of spinal muscular atrophy"
Results: 146
Healthcare Resource Utilization, Economic Burden, and Multi-Level Medical Security System for Individuals with Spinal Muscular Atrophy in Shaanxi Province, China.
- Published in:
- Healthcare (2227-9032), 2025, v. 13, n. 4, p. 428, doi. 10.3390/healthcare13040428
- By:
- Publication type:
- Article
An Assessment of the Knowledge, Attitudes, and Practices of Pediatricians and Pediatric Residents in Spinal Muscular Atrophy.
- Published in:
- Haydarpasa Numune Medical Journal, 2023, v. 6, n. 3, p. 231, doi. 10.14744/hnhj.2021.92260
- By:
- Publication type:
- Article
Rehabilitation in Spinal Muscular Atrophy: A Narrative Review.
- Published in:
- European Journal of Therapeutics, 2023, v. 29, n. 3, p. 656, doi. 10.58600/eurjther1516
- By:
- Publication type:
- Article
Genetic neuromuscular disorders: living the era of a therapeutic revolution. Part 2: diseases of motor neuron and skeletal muscle.
- Published in:
- 2019
- By:
- Publication type:
- journal article
Intrathecal administration of Nusinersen in type 1 SMA: successful psychological program in a single Italian center.
- Published in:
- 2018
- By:
- Publication type:
- journal article
Effects of electrical stimulation-induced leg training on skeletal muscle adaptability in spinal cord injury.
- Published in:
- Scandinavian Journal of Medicine & Science in Sports, 2002, v. 12, n. 5, p. 316, doi. 10.1034/j.1600-0838.2002.20106.x
- By:
- Publication type:
- Article
Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis.
- Published in:
- Frontiers in Molecular Neuroscience, 2017, p. 1, doi. 10.3389/fnmol.2017.00405
- By:
- Publication type:
- Article
Neuroprotective Effect of Non-viral Gene Therapy Treatment Based on Tetanus Toxin C-fragment in a Severe Mouse Model of Spinal Muscular Atrophy.
- Published in:
- Frontiers in Molecular Neuroscience, 2016, p. 1, doi. 10.3389/fnmol.2016.00076
- By:
- Publication type:
- Article
High-Frequency Chest-Wall Oscillation in a Noninvasive-Ventilation-Dependent Patient With Type 1 Spinal Muscular Atrophy.
- Published in:
- Respiratory Care, 2011, v. 56, n. 11, p. 1840, doi. 10.4187/respcare.01155
- By:
- Publication type:
- Article
Genetic therapy for spinal muscular atrophy.
- Published in:
- Nature Biotechnology, 2010, v. 28, n. 3, p. 235, doi. 10.1038/nbt0310-235
- By:
- Publication type:
- Article
Disease Mechanisms and Therapeutic Approaches in Spinal Muscular Atrophy.
- Published in:
- Journal of Neuroscience, 2015, v. 35, n. 23, p. 8691, doi. 10.1523/JNEUROSCI.0417-15.2015
- By:
- Publication type:
- Article
Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps.
- Published in:
- 2019
- By:
- Publication type:
- journal article
Table of Contents.
- Published in:
- Developmental Medicine & Child Neurology, 2017, v. 59, n. 8, p. 771, doi. 10.1111/dmcn.13272
- Publication type:
- Article
Long-term ventilation in spinal muscular atrophy: the price of progress.
- Published in:
- 2017
- By:
- Publication type:
- Editorial
News in brief.
- Published in:
- Indian Pediatrics, 2017, v. 54, n. 2, p. 167, doi. 10.1007/s13312-017-1024-3
- By:
- Publication type:
- Article
A qualitative study of perceptions of meaningful change in spinal muscular atrophy.
- Published in:
- 2017
- By:
- Publication type:
- journal article
Screening for spinal muscular atrophy.
- Published in:
- 2018
- By:
- Publication type:
- journal article
Antisense-based therapy for the treatment of spinal muscular atrophy.
- Published in:
- Journal of Cell Biology, 2012, v. 199, n. 1, p. 21, doi. 10.1083/jcb.201207087
- By:
- Publication type:
- Article
Spinal muscular atrophy type I and the dual role of viruses: An interview with Professor Basil T. Darras, Professor of Neurology (Pediatrics) at Harvard Medical School.
- Published in:
- Experimental & Therapeutic Medicine, 2018, v. 15, n. 4, p. 3673, doi. 10.3892/etm.2018.5884
- By:
- Publication type:
- Article
Spinal muscular atrophy, pediatric virology and gene therapy: A challenge of modern weakness and hope.
- Published in:
- Experimental & Therapeutic Medicine, 2018, v. 15, n. 4, p. 3671, doi. 10.3892/etm.2018.5883
- By:
- Publication type:
- Article
Application of urine cells in drug intervention for spinal muscular atrophy.
- Published in:
- Experimental & Therapeutic Medicine, 2017, v. 14, n. 3, p. 1993, doi. 10.3892/etm.2017.4791
- By:
- Publication type:
- Article
Spinal muscular atrophy disease: a literature review for therapeutic strategies.
- Published in:
- Journal of Medicine & Life, 2010, v. 3, n. 1, p. 3
- By:
- Publication type:
- Article
Spinal muscular atrophy.
- Published in:
- Journal of Paediatrics & Child Health, 2018, v. 54, n. 5, p. 589, doi. 10.1111/jpc.13912
- By:
- Publication type:
- Article
Eighteen Years with Spinal Muscular Atrophy (SMA) Type 1.
- Published in:
- 2013
- By:
- Publication type:
- Case Study
Tracheotomy and children with spinal muscular atrophy type 1: Ethical considerations in the French context.
- Published in:
- 2012
- By:
- Publication type:
- Journal Article
Recent Advances in Antisense Oligonucleotide Therapy in Genetic Neuromuscular Diseases.
- Published in:
- Annals of Indian Academy of Neurology, 2018, v. 21, n. 1, p. 3, doi. 10.4103/aian.AIAN_298_17
- By:
- Publication type:
- Article
Onasemnogene Abeparvovec: Post-infusion Efficacy and Safety in Patients With Spinal Muscular Atrophy (SMA)—A Fondazione Policlinico Gemelli IRCCS Experience.
- Published in:
- Hospital Pharmacy, 2024, v. 59, n. 1, p. 39, doi. 10.1177/00185787231182562
- By:
- Publication type:
- Article
Assays for the Identification and Prioritization of Drug Candidates for Spinal Muscular Atrophy.
- Published in:
- Assay & Drug Development Technologies, 2014, v. 12, n. 6, p. 315, doi. 10.1089/adt.2014.587
- By:
- Publication type:
- Article
Interview with Jill Jarecki, PhD.
- Published in:
- Assay & Drug Development Technologies, 2014, v. 12, n. 6, p. 311, doi. 10.1089/adt.2014.1505
- Publication type:
- Article
Does use of the Cough Assist Machine reduce respiratory morbidity for children with neuromuscular disease?
- Published in:
- New Zealand Journal of Physiotherapy, 2014, v. 42, n. 3, p. 126, doi. 10.15619/nzjp/42.3.03
- By:
- Publication type:
- Article
Nusinersen and sleep in children with spinal muscular atrophy.
- Published in:
- 2018
- Publication type:
- Abstract
Efficient gene expression from integration-deficient lentiviral vectors in the spinal cord.
- Published in:
- Gene Therapy, 2013, v. 20, n. 6, p. 645, doi. 10.1038/gt.2012.78
- By:
- Publication type:
- Article
Splice-Switching Therapy for Spinal Muscular Atrophy.
- Published in:
- Genes, 2017, v. 8, n. 6, p. 161, doi. 10.3390/genes8060161
- By:
- Publication type:
- Article
Management of Critically Ill Patients With Spinal Muscular Atrophy Admitted With Acute Respiratory Failure: The Authors' Reply.
- Published in:
- 2023
- By:
- Publication type:
- Letter to the Editor
A Paradigm Shift for the Management of Critically Ill Patients With Spinal Muscular Atrophy.
- Published in:
- 2023
- By:
- Publication type:
- Letter to the Editor
Management of Critically Ill Patients With Spinal Muscular Atrophy Admitted With Acute Respiratory Failure.
- Published in:
- Respiratory Care, 2023, v. 68, n. 2, p. 247, doi. 10.4187/respcare.10353
- By:
- Publication type:
- Article
Five-Year Follow-Up and Outcomes of Noninvasive Ventilation in Subjects With Neuromuscular Diseases.
- Published in:
- Respiratory Care, 2018, v. 63, n. 3, p. 274, doi. 10.4187/respcare.05475
- By:
- Publication type:
- Article
"...They were just treating her Symptom by Symptom": maternal experiences of having a child with spinal muscular atrophy in Ghana.
- Published in:
- BMC Palliative Care, 2025, v. 24, n. 1, p. 1, doi. 10.1186/s12904-025-01651-3
- By:
- Publication type:
- Article
Spinal Muscular Atrophy and Common Therapeutic Advances.
- Published in:
- Fetal & Pediatric Pathology, 2019, v. 38, n. 3, p. 226, doi. 10.1080/15513815.2018.1520374
- By:
- Publication type:
- Article
Treatment for Spinal Muscular Atrophy.
- Published in:
- Neurology Alert, 2017, v. 37, n. 4, p. 1
- By:
- Publication type:
- Article
Spinal Muscular Atrophy: Genetics And Recent Advancement In Gene Therapy.
- Published in:
- International Journal of High School Research, 2024, v. 6, n. 6, p. 61, doi. 10.36838/v6i6.10
- By:
- Publication type:
- Article
Science Digest.
- Published in:
- New Zealand Journal of Medical Laboratory Science, 2018, v. 72, n. 1, p. 29
- Publication type:
- Article
Transcriptional profiling of differentially vulnerable motor neurons at pre-symptomatic stage in the Smn<sup>2b/-</sup> mouse model of spinal muscular atrophy.
- Published in:
- Acta Neuropathologica Communications, 2015, v. 3, p. 1, doi. 10.1186/s40478-015-0231-1
- By:
- Publication type:
- Article
A Semi‐Mechanistic Population Pharmacokinetic Model of Nusinersen: An Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy.
- Published in:
- CPT: Pharmacometrics & Systems Pharmacology, 2018, v. 7, n. 9, p. 581, doi. 10.1002/psp4.12323
- By:
- Publication type:
- Article
Affording Genetic Therapies in the Medicaid Program.
- Published in:
- 2020
- By:
- Publication type:
- Opinion
Case of spinal muscular atrophy type 0 with mild prognosis.
- Published in:
- 2020
- By:
- Publication type:
- Case Study
Use of pediatric Tracheal Stoma Retainer<sup>®</sup> in a 24‐year‐old spinal muscular atrophy patient.
- Published in:
- 2018
- By:
- Publication type:
- Case Study
Clinical research advance of therapeutic strategies for spinal muscular atrophy.
- Published in:
- Chinese Journal of Contemporary Neurology & Neurosurgery, 2019, v. 19, n. 6, p. 385, doi. 10.3969/j.issn.1672-6731.2019.06.003
- By:
- Publication type:
- Article
Research progress of spinal muscular atrophy treatment in children.
- Published in:
- Chinese Journal of Contemporary Neurology & Neurosurgery, 2018, v. 18, n. 4, p. 284, doi. 10.3969/j.issn.1672-6731.2018.04.010
- By:
- Publication type:
- Article
Gene-Targeted Therapies and Palliative Care in Children with Spinal Muscular Atrophy Type I: No Intrinsic Contradiction.
- Published in:
- 2021
- By:
- Publication type:
- Letter