Clinical Trials

Learn About Clinical Trials - ClinicalTrials.gov

Learn About Clinical Trials - ClinicalTrials.gov

Recruiting - Gene Therapy for Pyruvate Kinase Deficiency (PKD) - ClinicalTrials.gov

Recruiting - Gene Therapy for Pyruvate Kinase Deficiency (PKD) - ClinicalTrials.gov

Recruiting - Pyruvate Kinase Deficiency Global Longitudinal Registry (PEAK Registry) - ClinicalTrials.gov

Recruiting - Pyruvate Kinase Deficiency Global Longitudinal Registry (PEAK Registry) - ClinicalTrials.gov

The Peak Registry is a global longitudinal study of patients diagnosed with pyruvate kinase deficiency, and is dedicated to understanding the full spectrum of disease, including impact on quality of life.

The Peak Registry is a global longitudinal study of patients diagnosed with pyruvate kinase deficiency, and is dedicated to understanding the full spectrum of disease, including impact on quality of life.

The Peak Registry - 1 page printable infographic

The Peak Registry - 1 page printable infographic

AG-348 (mitapivat), an allosteric activator of red blood cell pyruvate kinase, increases enzymatic activity, protein stability, and adenosine triphosphate levels over a broad range of PKLR genotypes, Haematologica 2021, Volume 106(1):238-249

AG-348 (mitapivat), an allosteric activator of red blood cell pyruvate kinase, increases enzymatic activity, protein stability, and adenosine triphosphate levels over a broad range of PKLR genotypes, Haematologica 2021, Volume 106(1):238-249

Trial Result - Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency New England Journal of Medicine September 5, 2019

Trial Result - Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency New England Journal of Medicine September 5, 2019

A Proposed Concept for Defective Mitophagy Leading to Late Stage Ineffective Erythropoiesis in Pyruvate Kinase Deficiency - van Vuren AJ, van Beers EJ and van Wijk R (2021) A Proposed Concept for Defective Mitophagy Leading to Late Stage Ineffective

A Proposed Concept for Defective Mitophagy Leading to Late Stage Ineffective Erythropoiesis in Pyruvate Kinase Deficiency - van Vuren AJ, van Beers EJ and van Wijk R (2021) A Proposed Concept for Defective Mitophagy Leading to Late Stage Ineffective Erythropoiesis in Pyruvate Kinase Deficiency. Front. Physiol. 11:609103. doi: 10.3389/fphys.2020.609103

Advocating for Yourself or Your Child in a Clinical Trial - Dr. Mark Sorrentino, a physician who has participated in a clinical trial & Dr. Joanna L. Perkins, a physician with experience as a principal investigator in clinical research, share their p

Advocating for Yourself or Your Child in a Clinical Trial - Dr. Mark Sorrentino, a physician who has participated in a clinical trial & Dr. Joanna L. Perkins, a physician with experience as a principal investigator in clinical research, share their perspectives on advocating for yourself or your child when participating in a clinical trial.