Works matching DE "TREATMENT of spinal muscular atrophy"
Results: 146
Spinal Muscular Atrophy: Genetics And Recent Advancement In Gene Therapy.
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- International Journal of High School Research, 2024, v. 6, n. 6, p. 61, doi. 10.36838/v6i6.10
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- Article
Rehabilitation in Spinal Muscular Atrophy: A Narrative Review.
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- European Journal of Therapeutics, 2023, v. 29, n. 3, p. 656, doi. 10.58600/eurjther1516
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- Article
Antisense-based therapy for the treatment of spinal muscular atrophy.
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- Journal of Cell Biology, 2012, v. 199, n. 1, p. 21, doi. 10.1083/jcb.201207087
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- Article
Segmental Spinal Muscular Atrophy Localised to the Lower Limbs: First case from Oman.
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- Sultan Qaboos University Medical Journal, 2017, v. 17, n. 3, p. 355, doi. 10.18295/squmj.2017.17.03.018
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- Article
Splice-Switching Therapy for Spinal Muscular Atrophy.
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- Genes, 2017, v. 8, n. 6, p. 161, doi. 10.3390/genes8060161
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- Article
Ventilator use in progressive neuromuscular disease: impact on patients and their families.
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- 1988
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- journal article
News in brief.
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- Indian Pediatrics, 2017, v. 54, n. 2, p. 167, doi. 10.1007/s13312-017-1024-3
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- Article
Screening for spinal muscular atrophy.
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- 2018
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- journal article
Conventional JOA score for cervical myelopathy has a rater's bias -In comparison with JOACMEQ.
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- 2018
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- journal article
Does pre-operative magnetic resonance imaging of the lumbar multifidus muscle predict clinical outcomes following lumbar spinal decompression for symptomatic spinal stenosis?
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- 2017
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- journal article
Association between paraspinal muscle morphology, clinical symptoms and functional status in patients with lumbar spinal stenosis.
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- 2017
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- journal article
Onasemnogene Abeparvovec (AVXS-101) for the Treatment of Spinal Muscular Atrophy.
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- Journal of Pediatric Pharmacology & Therapeutics, 2021, v. 26, n. 5, p. 437, doi. 10.5863/1551-6776-26.5.437
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- Article
Gene-Targeted Therapies and Palliative Care in Children with Spinal Muscular Atrophy Type I: No Intrinsic Contradiction.
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- 2021
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- Letter
The Role of Chest Physiotherapy in Prevention of Postextubation Atelectasis in Pediatric Patients with Neuromuscular Diseases.
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- Iranian Journal of Child Neurology, 2013, v. 7, n. 1, p. 21
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- Article
Roche to acquire Spark Therapeutics [including commentary by Pravin U. Dugel].
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- Ocular Surgery News, 2019, v. 37, n. 6, p. 1
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- Article
High-Frequency Chest-Wall Oscillation in a Noninvasive-Ventilation-Dependent Patient With Type 1 Spinal Muscular Atrophy.
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- Respiratory Care, 2011, v. 56, n. 11, p. 1840, doi. 10.4187/respcare.01155
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- Article
Symptom management and psychological support for families are the cornerstones of end-of-life care for children with spinal muscular atrophy type 1.
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- 2018
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- journal article
Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy - a literature review.
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- 2017
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- journal article
Disease burden of spinal muscular atrophy in Germany.
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- 2016
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- journal article
Disease Mechanisms and Therapeutic Approaches in Spinal Muscular Atrophy.
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- Journal of Neuroscience, 2015, v. 35, n. 23, p. 8691, doi. 10.1523/JNEUROSCI.0417-15.2015
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- Article
Spinal Muscular Atrophy and Common Therapeutic Advances.
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- Fetal & Pediatric Pathology, 2019, v. 38, n. 3, p. 226, doi. 10.1080/15513815.2018.1520374
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- Article
Intrathecal administration of nusinersen in adolescent and adult SMA type 2 and 3 patients.
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- Journal of Neurology, 2019, v. 266, n. 1, p. 183, doi. 10.1007/s00415-018-9124-0
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- Article
Transcriptional profiling of differentially vulnerable motor neurons at pre-symptomatic stage in the Smn<sup>2b/-</sup> mouse model of spinal muscular atrophy.
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- Acta Neuropathologica Communications, 2015, v. 3, p. 1, doi. 10.1186/s40478-015-0231-1
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- Article
Reversal of neuromuscular blockade with sugammadex in a patient with spinal muscular atrophy type III (Kugelberg-Welander syndrome).
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- 2012
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- Publication type:
- Letter
Nusinersen: First Global Approval.
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- Drugs, 2017, v. 77, n. 4, p. 473, doi. 10.1007/s40265-017-0711-7
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- Article
Odontoid Non Union with Myelopathy - "Joint Jamming Procedure" with Posterior C1-C2 Lateral Mass Fusion.
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- 2012
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- Case Study
Tracheotomy and children with spinal muscular atrophy type 1: Ethical considerations in the French context.
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- 2012
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- Publication type:
- Journal Article
Therapy Development for Spinal Muscular Atrophy in SMN Independent Targets.
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- Neural Plasticity, 2012, v. 2012, p. 1, doi. 10.1155/2012/456478
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- Article
Current Status of Treatment of Spinal and Bulbar Muscular Atrophy.
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- Neural Plasticity, 2012, v. 2012, p. 1, doi. 10.1155/2012/369284
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- Article
Increase of hyperpolarization-activated cyclic nucleotide-gated current in the aberrant excitability of spinal muscular atrophy.
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- 2018
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- journal article
Emerging therapies and challenges in spinal muscular atrophy.
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- 2017
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- journal article
Social Media as a Source of Knowledge about Gene Therapy for Spinal Muscular Atrophy.
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- Healthcare (2227-9032), 2022, v. 10, n. 12, p. 2445, doi. 10.3390/healthcare10122445
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- 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.
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- Experimental & Therapeutic Medicine, 2018, v. 15, n. 4, p. 3673, doi. 10.3892/etm.2018.5884
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- Article
Spinal muscular atrophy, pediatric virology and gene therapy: A challenge of modern weakness and hope.
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- Experimental & Therapeutic Medicine, 2018, v. 15, n. 4, p. 3671, doi. 10.3892/etm.2018.5883
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- Article
Application of urine cells in drug intervention for spinal muscular atrophy.
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- Experimental & Therapeutic Medicine, 2017, v. 14, n. 3, p. 1993, doi. 10.3892/etm.2017.4791
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- Article
Late adult-onset pure spinal muscular atrophy due to a compound HEXB macro-deletion.
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- Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration, 2013, v. 14, n. 7/8, p. 628, doi. 10.3109/21678421.2013.812662
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- Article
Feasibility of Using Microsoft Kinect to Assess Upper Limb Movement in Type III Spinal Muscular Atrophy Patients.
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- PLoS ONE, 2017, v. 12, n. 1, p. 1, doi. 10.1371/journal.pone.0170472
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- Article
Activin Receptor Type IIB Inhibition Improves Muscle Phenotype and Function in a Mouse Model of Spinal Muscular Atrophy.
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- PLoS ONE, 2016, v. 11, n. 11, p. 1, doi. 10.1371/journal.pone.0166803
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- Article
Spontaneous Breathing Pattern as Respiratory Functional Outcome in Children with Spinal Muscular Atrophy (SMA).
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- PLoS ONE, 2016, v. 11, n. 11, p. 1, doi. 10.1371/journal.pone.0165818
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- Article
Altered mRNA Splicing in SMN-Depleted Motor Neuron-Like Cells.
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- PLoS ONE, 2016, v. 11, n. 10, p. 1, doi. 10.1371/journal.pone.0163954
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- Article
A spinalis izomatrophia felismerésének fontossága.
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- Lege Artis Medicine (LAM), 2022, v. 32, n. 4/5, p. 207, doi. 10.33616/lam.32.018
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- Article
IT'S NOT THE TIME TO RELAX ON PEDIATRIC MEDICINES R&D.
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- Applied Clinical Trials, 2018, v. 27, n. 12, p. 7
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- Article
Call in the backup.
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- Nature Medicine, 2012, v. 18, n. 11, p. 1602, doi. 10.1038/nm1112-1602
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- Article
Viral delivery of miR-196a ameliorates the SBMA phenotype via the silencing of CELF2.
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- Nature Medicine, 2012, v. 18, n. 7, p. 1136, doi. 10.1038/nm.2791
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- Article
Onasemnogene Abeparvovec: Post-infusion Efficacy and Safety in Patients With Spinal Muscular Atrophy (SMA)—A Fondazione Policlinico Gemelli IRCCS Experience.
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- Hospital Pharmacy, 2024, v. 59, n. 1, p. 39, doi. 10.1177/00185787231182562
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- Article
A qualitative study of perceptions of meaningful change in spinal muscular atrophy.
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- 2017
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- Publication type:
- journal article
Bilateral aberrant C1/2 intradural vertebral arteries: a rare cause of cervical myelopathy.
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- ANZ Journal of Surgery, 2017, v. 87, n. 3, p. 202, doi. 10.1111/ans.12870
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- Article
Abstracts for the 42nd Human Genetics Society of Australasia Annual Scientific Meeting Sydney, New South Wales August 4-7, 2018.
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- 2018
- Publication type:
- Abstract
Beta-lactam antibiotic offers neuroprotection in a spinal muscular atrophy model by multiple mechanisms.
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- Annals of Neurosciences, 2011, v. 18, n. 4, p. 156, doi. 10.5214/ans.0972.7531.1118407
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- Article
Nutritional, Gastrointestinal and Endo-Metabolic Challenges in the Management of Children with Spinal Muscular Atrophy Type 1.
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- Nutrients, 2021, v. 13, n. 7, p. 2400, doi. 10.3390/nu13072400
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- Publication type:
- Article