Here we define and review endpoints for measuring AED treatment success and explore the concepts of efficacy, tolerability, QOL, retention, and compliance as well as their usefulness as clinical trial endpoints.
We believe that these data provide proof-of-concept of efficacy of the proposed combined treatments in preserving PNS and CNS functionality in Twi mice.
This study gives proof-of-concept of efficacy, tolerability and clinical relevance of the combined gene/cell therapies proposed here, which may constitute a feasible and effective therapeutic opportunity for children affected by GLD.
In this work, we provide proof-of-concept of efficacy, tolerability and clinical relevance of combining CNS-directed approaches (transplant of gene-corrected NSCs or LV-based IC GT) with BMT to correct the GLD phenotype in a mouse model that recapitulates the severity and rapid progression of the early infantile human disease.
On the basis of broad in vitro and in vivo proof of concept for efficacy of telomerase inhibition in many major cancer types tested, good safety profile and excellent pharmacokinetics and bio-distribution, GRN163L has entered clinical trials (Geron Corporation).
The first proof of concept test of efficacy of an RNAi-based therapeutic in man has been initiated.
Using the concept of stoichiometric efficacy, we determined that I-F11 affords protection from lethality against an administered dose of 10, 15, 90 and 80 molar equivalents of GA, GB, GD and GF, respectively, relative to the molar equivalents of I-F11 in circulation.
However, the concept of 50% efficacy was seen as a weakness of the product.
Finally, in human and veterinary preclinical study, considering the high incidence of naturally occurring OSA in dogs, this mouse model could be used as a tool, to obtain a proof of concept of the efficacy of novel therapeutic approaches.
42 The concept of maximizing efficacy and minimizing adverse side effects through the combined use of topical, phototherapy, and systemic agents has been exercised extensively.
