New member of the expert board – MUDr. Alena Zumrová, Ph.D

We are incredibly honoured to welcome a new addition to our team on the expert board. We greatly appreciate the doctor for her lifetime of experience in neurology and her willingness to support patient efforts.

Our core mission is to bridge the worlds of science, medicine and patients. Thanks to the doctor, that will be a little easier again. As a first goal, she and I set out to find out the current real number of patients with Angelman syndrome in the Czech Republic. If you were surprised that such an elementary figure is missing, it is indeed a sad fact. There is no institution in the Czech Republic that is obliged to publish such data. However, knowing the most accurate number of patients is essential for any further action. All in accordance with GDPR, of course, we are concerned with an anonymised aggregate count of all patients like Oliver.

Doctor, welcome to the team. We’re really excited about this.

MUDr. Alena Zumrová, Ph.D.

She has been working since 1982 at the Department of Child Neurology at the 2nd Faculty of Medicine, Charles University in Prague and Motol University Hospital in Prague.In 2008, she founded the Motol University Hospital Hereditary Ataxia Centre with nationwide coverage, which in 2018 was awarded the status of a reference centre within the European Reference Network for Rare Neurological Diseases (ERN-RND). It focuses on neurodevelopmental, neurometabolic, neurodegenerative and neurogenetic rare diseases.

What are cell and gene therapies and how do they work?

Transformative cell and gene therapies represent a new frontier of medicine.They offer hope for many patients, particularly those affected by rare diseases. Gene therapies are a success story of the 21st century, with exciting treatment options in the pipeline for many diseases, such as rare bleeding disorders or different forms of neuromuscular disorders. Likewise, cell-based products are offering new hope for several conditions (e.g. CAR-Ts for certain blood cancers).

Cell and gene therapies have fundamentally different properties than medicines and surgery in that they treat the core underlying genetic causes of a disease rather than treating symptoms.


Gene therapy is the use of genetic material to treat genetic diseases. This may involve adding or replacing a wild type copy of the gene (gene addition or gene replacement) or altering a gene with mutation to the wild type gene (gene editing). The treatment may take place outside of the body (ex vivo) or inside the body (in vivo).


Cell therapy is the transfer of intact, live cells into a patient to help lessen or cure a disease. The cells may originate from the patient (autologous cells) or a donor (allogeneic cells). The cells used in cell therapy can be classified by their potential to transform into different cell types. Pluripotent cells can transform into any cell type in the body and multipotent cells can transform into other cell types, but their repertoire is more limited than that of pluripotent cells.

Introducing our partner – the international company DNV GL

DNV GL is global independent services provider in the area of risk management, certification, validation, verification and trainings in all industry sectors with a special focus on healthcare.

Last year company DNV GL decided to support our nonprofit organisation ASGENT.ORG. We regard this help a lot not only as substantial financial support but more as expression of support and trust on the beginning of our long journey. It is most important to know that our efforts make sense and is supported by such important and respected partner.

We thanks especially to the Regional Manager of Business Assurance for Central Europe represented by Mr. Shahram Ghaem Maralani, and then thanks to the Executive Director for the Czech and Slovak Republics Mrs. Maria Lichnerová and the Sales Director for the Czech and Slovak Republics Mrs. Zdenka Šimková. THANK YOU