Spinal Muscular Atrophy — Respiratory Muscle Training in Patients With Spinal Muscular Atrophy (SMA).
Citation(s)
Aslan GK, Gurses HN, Issever H, Kiyan E Effects of respiratory muscle training on pulmonary functions in patients with slowly progressive neuromuscular disease: a randomized controlled trial. Clin Rehabil. 2014 Jun;28(6):573-81. doi: 10.1177/0269215513512215. Epub 2013 Nov 25.
Finkel RS, Sejersen T, Mercuri E; ENMC SMA Workshop Study Group 218th ENMC International Workshop:: Revisiting the consensus on standards of care in SMA Naarden, The Netherlands, 19-21 February 2016. Neuromuscul Disord. 2017 Jun;27(6):596-605. doi: 10.1016/j.nmd.2017.02.014. Epub 2017 Mar 2. No abstract available.
Human A, Morrow BM Inspiratory muscle training in children and adolescents living with neuromuscular diseases: A pre-experimental study. S Afr J Physiother. 2021 Aug 31;77(1):1577. doi: 10.4102/sajp.v77i1.1577. eCollection 2021.
Koopman M, Zanen P, Kruitwagen CL, van der Ent CK, Arets HG Reference values for paediatric pulmonary function testing: The Utrecht dataset. Respir Med. 2011 Jan;105(1):15-23. doi: 10.1016/j.rmed.2010.07.020. Erratum in: Respir Med. 2011 Dec;105(12):1970-1.
Paul GR, Gushue C, Kotha K, Shell R The respiratory impact of novel therapies for spinal muscular atrophy. Pediatr Pulmonol. 2021 Apr;56(4):721-728. doi: 10.1002/ppul.25135. Epub 2020 Nov 2.
Quanjer PH, Tammeling GJ, Cotes JE, Pedersen OF, Peslin R, Yernault JC Lung volumes and forced ventilatory flows. Report Working Party Standardization of Lung Function Tests, European Community for Steel and Coal. Official Statement of the European Respiratory Society. Eur Respir J Suppl. 1993 Mar;16:5-40. No abstract available.
Wadman RI, Vrancken AF, van den Berg LH, van der Pol WL Dysfunction of the neuromuscular junction in spinal muscular atrophy types 2 and 3. Neurology. 2012 Nov 13;79(20):2050-5. doi: 10.1212/WNL.0b013e3182749eca. Epub 2012 Oct 31.
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.