Expert Interviews

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.

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Professor Eyal Grunebaum and Professor Michael Hershfield discuss advances in the management of ADA-SCID, looking at a range of factors that have influenced change, including enzyme replacement therapy, other therapeutic options, and the importance of genotype/phenotype correlations, and look towards the future with an optimistic outlook for ADA-SCID patients.

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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.

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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.

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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.

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