• Home Agios begins AG-348 Phase 1 study to treat PK deficiency Agios Pharmaceuticals.

Agios begins AG-348 Phase 1 study to treat PK deficiency Agios Pharmaceuticals.

‘Agios believes in the potential of small molecules to become disease-modifying therapeutics for rare genetic disorders by targeting the specific metabolic defects generating these diseases. We anticipate that the results out of this healthy volunteer research will enable us to move AG-348 rapidly into a research in patients with PK insufficiency.’ ‘Preclinical studies possess demonstrated that AG-348 activates a wide spectrum of PK-R mutant proteins, and corrects the metabolic defects within patient-derived blood samples,’ said Scott Biller, chief scientific officer of Agios. ‘These data support the hypothesis that medication intervention with AG-348 will restore glycolytic pathway flux and normalize reddish blood cell metabolism.Seventy-six % of the kids had normal serum IgE levels, a proportion similar to that previously reported among children with transient wheezing.6 Despite our greatest intentions, we inadvertently included nine children in whom persistent or atopic asthma symptoms developed through the study period. Third, we examined parents’ initiation of preemptive treatment with fluticasone since it would happen in real-life practice. Due to the perceived importance of treatment early in the course of an upper respiratory system contamination, the intervention was based on parental perception of the current presence of an upper respiratory tract infection, instead of on objective documentation of infections or a specified minimal duration of symptoms.41 Although a viral trigger has been confirmed in 74 percent of top respiratory tract attacks identified by parents,42 we can not rule out feasible misidentification of colds.