Episode 135

Patient Perspectives on Gene Therapy for Sickle Cell Disease and B-Thalassemia

Join us for an insightful interview with patient advocates Miriam Santos Freire (sickle cell disease) and Loris Brunetta (beta-thalassemia major). Benefit from their experiences as they discuss with our host Isabel Olivera-Martinez the promises and challenges of gene therapy for these conditions.

Listen to their experiences of living with these genetic conditions while they highlight the disparities in treatment access between wealthy and low-income countries. Both view gene therapy as a breakthrough that has the potential to cure these diseases, but express caution regarding its current limitations and complications. Tune in for their valuable insights and recommendations for advancing SCD and thalassemia management.

Host: Isabel Olivera-Martinez, PhD, Medical Writer 

Guests:

Miriam Santos Freire, Patient representative for EuroBloodNet

Patient Advocacy & Communication for Sickle Cell (Portugal & UK) at European Sickle Cell Federation (ESCF)

European Hematology Association (EHA)

ERN-EuroBloodNet

Associação Portuguesa de Pais de Doentes com Hemoglobinopatias (APPDH) – also a Board Member

Loris Brunetta

Board Member of Thalassaemia International Federation (TIF),

Vice-chair of EHA Patients’ Advocacy Committee (PAC),

Member of the Patients and Consumer Working Party (PCWP) at EMA,

EMA expert on ESEC,