New survey! Expectations/requirements for a potential gene editing for Pyruvate Kinase Deficiency (PKD) and Congenital Dyserythropoietic Anemia (CDA)
This survey is designed to gather insights into patients' expectations and requirements for potential gene therapy treatments for Pyruvate Kinase Deficiency (PKD) and Congenital Dyserythropoietic Anemia (CDA).
To better understand the expectations, concerns, and requirements of patients affected by Pyruvate Kinase Deficiency (PKD) and Congenital Dyserythropoietic Anemia Type II (CDAII) regarding gene therapy, the Cell Technology Division from CIEMAT/CIBERER/IIS-FDJ and ERN-EuroBloodNet is conducting a dedicated survey targeting patients and their families.
This survey explores key topics such as awareness of gene therapy, interest in future treatment options, and willingness to participate in future gene therapy clinical trials. The responses collected will help inform the development of patient-centered communication strategies and guide the design of future therapeutic approaches.
Preliminary results will be presented during the upcoming Patients Webinar on Gene Therapy for PKD and CDAII, providing a unique opportunity to connect cutting-edge research with real-world patient perspectives.
To access the survey, please use the following links, which are available in different languages:
This anonymous survey will take less than 10 minutes to complete, and your answers will help us develop better gene therapy for treating these diseases.