Dursun N, Gokbel T, Akarsu M, Dursun E Randomized Controlled Trial on Effectiveness of Intermittent Serial Casting on Spastic Equinus Foot in Children with Cerebral Palsy After Botulinum Toxin-A Treatment. Am J Phys Med Rehabil. 2017 Apr;96(4):221-225. doi: 10.1097/PHM.0000000000000627.
Nguyen L, Di Rezze B, Mesterman R, Rosenbaum P, Gorter JW Effects of Botulinum Toxin Treatment in Nonambulatory Children and Adolescents With Cerebral Palsy: Understanding Parents' Perspectives. J Child Neurol. 2018 Oct;33(11):724-733. doi: 10.1177/0883073818786567. Epub 2018 Jul 24.
Nguyen L, Mesterman R, Gorter JW Development of an inventory of goals using the International Classification of Functioning, Disability and Health in a population of non-ambulatory children and adolescents with cerebral palsy treated with botulinum toxin A. BMC Pediatr. 2018 Jan 4;18(1):1. doi: 10.1186/s12887-017-0974-x.
Reid SM, Carlin JB, Reddihough DS Using the Gross Motor Function Classification System to describe patterns of motor severity in cerebral palsy. Dev Med Child Neurol. 2011 Nov;53(11):1007-12. doi: 10.1111/j.1469-8749.2011.04044.x. Review.
Integrated Botulinum Toxin for Improved Sitting Balance in Children With Cerebral Palsy: Body Functions to Activity and Participation
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.
