Pioneering progress in ADA-SCID with gene therapy
Strimvelis ▼ (Autologous CD34+ cells transduced to express ADA) is indicated for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)- matched related stem cell donor is available (see Summary of Product Characteristics for more information).
Click here for the full Strimvelis PI.
GSK in rare diseases
Dr Sven Kili
ADA-SCID: today and tomorrow
Dr Pauline Ng
Overview of HSCT and ADA-SCID: current treatments and outcomes
Dr Andrew Gennery
Ex vivo gene therapy in ADA-SCID: clinical data and experiences to date
Professor Alessandro Aiuti
Role of the referrer and the overall treatment pathway – a direct experience
Dr Robbert Bredius
The healthcare professional referrer roadmap
Dr Pauline Ng
The videos presented above contain promotional content on Strimvelis .
Strimvelis is not marketed in all EU countries.
The Full Prescribing Information is available in English here.
The French Summary of Product Characteristics is available here.
▼ This medical product is subject to additional monitoring. This will allow early identification of new safety information. Health Care Professionals are encouraged to report any suspected adverse reactions to their local GSK office and their national reporting system (for France: Agence Nationale de Sécurité du Médicament et des produits de santé - http://www.ansm.sante.fr).
ADA-SCID, adenosine deaminase severe combined immunodeficiency; HSCT, haematopoietic stem cell transplantation.