Sentences with phrase «optimal efficacy and safety»

Amgen's approach is to understand the fundamental biological mechanisms of disease in order to determine what type of medicine is most likely to deliver optimal efficacy and safety.

Not exact matches

«This Phase 1 trial in children with SMA Type 2 will allow us to evaluate safety, optimal dosing and proof - of - concept for efficacy of AVXS - 101 compared to the well - characterized natural history using the one - time intrathecal route of administration,» said Dr. Sukumar Nagendran, Chief Medical Officer of AveXis.

U.S. Phase 1 Trial in SMA Type 2 (STRONG) The open - label, dose - comparison, multi-center Phase 1 trial — known as STRONG — is designed to evaluate the safety, optimal dosing, and proof of concept for efficacy of AVXS - 101 in two distinct age groups of patients with SMA Type 2, utilizing a one - time IT route of administration.

It is at present unknown whether an optimal balance between safety and efficacy can be achieved with the combination therapy of GH and IGF - I, since this combination has been evaluated in only a small number of patient populations and in studies of a relatively short duration.

Research related to optimal youth development has begun to delineate critical dimensions of important social contexts.8 — 11 For example, some researchers propose 3 basic experiences (ie, connection, regulation, and autonomy) to define youth's main associations with their environment that can be measured across multiple settings.8, 12,13 Others have articulated similar concepts and expanded the number of dimensions to include characteristics, such as adequate nutrition, health, and shelter; physical and psychological safety; adequate and appropriate supervision and motivation; supportive relationships; support for efficacy and opportunities for skill building; and integration of family, school, and community efforts.10, 11