By Bernard Ravina; et al

''Translating laboratory discoveries into profitable therapeutics could be tough. medical Trials in Neurology goals to enhance the potency of medical trials and the advance of interventions in an effort to improve the advance of latest remedies for neurologic illnesses. It introduces the reader to the foremost suggestions underpinning trials within the neurosciences. This quantity tackles the demanding situations of developing Read more...

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Some of these problems have arisen because we have historically used clinical scales that were developed to describe subgroups of domains afected by CNS diseases. One example is the EDSS in MS which is largely inluenced by lower extremity walking function, but less so by cognition or upper extremity function. Furthermore, it has been extremely diicult to determine and agree on what degree of change represents a clinically meaningful outcome. All of these factors have contributed to a high degree of failure in late stages of development (see Chapter 2).

Blood-brain barrier active elux transporters: ATP-binding cassette gene family. NeuroRx 2005; 2: 86–98. 16. Patel MM, Goyal BR, Bhadada SV, et al. Getting into the brain: approaches to enhance brain drug delivery. CNS Drugs 2009; 23: 35–58. 17. Neuwelt E, Abbott NJ, Abrey L, et al. Strategies to advance translational research into brain barriers. Lancet Neurol 2008; 7: 84–96. Chapter 3: Unique challenges in the development of therapies 18. Rambeck B, Jurgens UH, May TW, et al. Comparison of brain extracellular luid, brain tissue, cerebrospinal luid, and serum concentrations of antiepileptic drugs measured intraoperatively in patients with intractable epilepsy.

32. Weinmann O, Schnell L, Ghosh A, et al. Intrathecally infused antibodies against Nogo-A penetrate the CNS and downregulate the endogenous neurite growth inhibitor Nogo-A. Mol Cell Neurosci 2006; 32: 161–73. 33. Brooks DJ, Papapetropoulos S, Vandenhende F, et al. An open-label, positron emission tomography study to assess adenosine A2A brain receptor occupancy of vipadenant (BIIB014) at steady-state levels in healthy male volunteers. Clin Neuropharmacol 2010; 33: 55–60. 34. Finkel RS. Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124).

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