Spinal Muscular Atrophy — Spinal Muscular Atrophy on Wheels, Using Power Mobility
Citation(s)
Arnold WD, Kassar D, Kissel JT Spinal muscular atrophy: diagnosis and management in a new therapeutic era. Muscle Nerve. 2015 Feb;51(2):157-67. doi: 10.1002/mus.24497. Epub 2014 Dec 16. Review.
Demers L, Weiss-Lambrou R, Ska B Item analysis of the Quebec User Evaluation of Satisfaction with Assistive Technology (QUEST). Assist Technol. 2000;12(2):96-105.
Di Marino E, Tremblay S, Khetani M, Anaby D The effect of child, family and environmental factors on the participation of young children with disabilities. Disabil Health J. 2018 Jan;11(1):36-42. doi: 10.1016/j.dhjo.2017.05.005. Epub 2017 Jun 1.
Feldner HA, Logan SW, Galloway JC Why the time is right for a radical paradigm shift in early powered mobility: the role of powered mobility technology devices, policy and stakeholders. Disabil Rehabil Assist Technol. 2016 Feb;11(2):89-102. doi: 10.3109/
Krasny-Pacini A, Hiebel J, Pauly F, Godon S, Chevignard M Goal attainment scaling in rehabilitation: a literature-based update. Ann Phys Rehabil Med. 2013 Apr;56(3):212-30. doi: 10.1016/j.rehab.2013.02.002. Epub 2013 Feb 28. Review.
Livingstone R, Field D Systematic review of power mobility outcomes for infants, children and adolescents with mobility limitations. Clin Rehabil. 2014 Oct;28(10):954-64. doi: 10.1177/0269215514531262. Epub 2014 Apr 24. Review.
Ostensjø S, Carlberg EB, Vøllestad NK The use and impact of assistive devices and other environmental modifications on everyday activities and care in young children with cerebral palsy. Disabil Rehabil. 2005 Jul 22;27(14):849-61.
St John BM, Hladik E, Romaniak HC, Ausderau KK Understanding health disparities for individuals with intellectual disability using photovoice. Scand J Occup Ther. 2018 Sep;25(5):371-381. doi: 10.1080/11038128.2018.1502349. Epub 2018 Oct 3.
Community's Participation of Children With Spinal Muscular Atrophy Using Power Mobility
Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.
Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.
Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.
Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.
Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.
