All posts by jcoh888

PK Deficiency Patient & Family Conference

Agios Pharmaceuticals will be hosting a Pyruvate Kinase Deficiency Patient and Family Conference in Millbrae, California on Saturday, November 4th.

This free event will be a unique opportunity to hear from scientific and medical experts who are leading the way in Pyruvate Kinase Deficiency research. In addition, patients and families will have time to connect, share stories, and support one another.

To learn more about this upcoming conference and/or to register, click here.





Patient Interview Study

Agios is developing a questionnaire that will help them to understand the impact of PK deficiency on both patients and caregivers.  To learn this information, a trained interviewer will be conducting face to face interviews with adults, adolescents, and young children with PK deficiency, and caregivers of children with PK deficiency.  Continue reading Patient Interview Study

European Symposium on Rare Anaemias

The 6th European Symposium on Rare Anaemias is quickly approaching. Organized by the European Network for Rare and Congenital Anaemias, this first Dutch-Belgium meeting for patients and health professionals will take place November 21-22, 2015 in Amsterdam, The Netherlands. This event is of no cost to patients and their families and will be presented in Dutch and English. Continue reading European Symposium on Rare Anaemias

Agios launches the DRIVE PK Trial website

Agios has launched a new website called Drive PK trial, which offers patients, caregivers, and healthcare professionals information about their phase 2 clinical trial for the oral medication, AG-348. This medicine has been developed as a potential treatment for pyruvate kinase deficiency.  Due to a deficiency in the enzyme, pyruvate kinase, people with pk deficiency are anemic, which means they have a reduced number of healthy red blood cells.  The study is being conducted to determine Continue reading Agios launches the DRIVE PK Trial website

Clinical Trial of AG-348 for Patients with PK Deficiency

The clinical study designed to evaluate the safety and efficacy of Agios Pharmaceutical’s AG-348 in adult patients with pyruvate kinase deficiency has opened at Boston Children’s Hospital.  This is the first site of a multicenter study that will open in North America and Europe.

Childrens Hospital-boston

AG-348 is an oral medication being studied as a possible treatment for PK deficiency. Researchers believe that this medicine may allow a patient with pyruvate kinase deficiency to maintain a normal amount of healthy red blood cells, and avoid other complications associated with this disease. Continue reading Clinical Trial of AG-348 for Patients with PK Deficiency