Defining Gene Therapy for Patients

Gene therapy is a welcome treatment for patients, which has been anticipated for decades. But how do parents and patients discuss this with their HTC staff, when they don’t know the language of science associated with gene therapy? A bleeding disorder gene therapy lexicon is currently being developed by various industry stakeholders, which is highly needed for dissemination of the complexities of gene therapy to patients, caregivers, and healthcare providers. Read MASAC Document 285.

Source: National Bleeding Disorders Foundation, August 2024