Watch our team of leading experts provide their insights into the challenges facing Healthcare Professionals in understanding and diagnosing patients with adenosine deaminase severe combined immunodeficiency (ADA-SCID) and discussing current and emerging treatments.
Professor Marina Cavazzana and Professor Arjan Lankester discuss techniques for newborn screening using tandem mass spectrometry, when to stop enzyme-replacement therapy before HSCT and gene therapy treatment, ADA deficiency as a multi organ disease and changes in the treatment algorithm for ADA-SCID.WATCH INTERVIEW
Dr Andrew Gennery discusses the challenges facing specialists in treating ADA-SCID, current and future treatments, and the need for a comprehensive central resource for clinicians to obtain up-to-date information about the disease.WATCH INTERVIEW
Professor Bobby Gaspar discusses the importance of identification methods to detect ADA-SCID patients, considerations when choosing the preferable current and future treatment options, gene therapy trial success and the need to share high-level expertise across the paediatric immunology community.WATCH INTERVIEW
Dr Robbert Bredius discusses early diagnosis of ADA-SCID, unique characteristics of the disease, the need for familial education and the requirement for physicians to understand the options for various treatment pathways.WATCH INTERVIEW