Press Releases King Baudouin Foundation: 28/2 International Rare Disease Day

King Baudouin Foundation: 28/2 International Rare Disease Day

Gene therapy offers significant promise for patients with rare diseases

Thousands of people in Belgium are living with rare diseases, which often cause serious physical and/or mental disabilities or may even be life-threatening. On International Rare Disease Day we focus on those people and the research that takes place into their diseases.

Extensive progress has been made in recent decades in understanding rare diseases and working towards treatments. Many rare diseases are caused by genetic defects, so gene therapy is very promising: repairing or replacing the affected gene would significantly slow down or stop the progress of the disease.

Prof. Thierry VandenDriessche (VUB) explains how it works in this article. He has been specialising in gene therapy for thirty years, and is currently working on three rare muscle diseases in particular – Duchenne’s muscular dystrophy, Steinert disease and Pompe’s disease.

Prof. VandenDriessche emphasises the role of patient associations, which challenge researchers to find out more about their disease and also raise funds for them.

This observation is quite well-known to us. The King Baudouin Foundation manages 14 Funds that are working on research into a specific rare disease, a group of rare diseases or rare diseases in general. Patients and/or those close to them are often the driving force behind this: they work tirelessly to get rare diseases onto the research agenda, help provide funding for research, often participate in clinical trials and follow the results closely. Finally, they work hard to ensure that treatments are made available.

Leading scientists, who are also unrelenting in their quests, support them in this. We would like to mention once again the Generet Prize for Rare Diseases, which was awarded in late January to Prof. Pierre Vanderhaeghen (KULeuven-VIB) for his research into human brain development and the things that can go wrong in rare diseases. The new call for the Generet Prize is now open for applications

Erika Racquet
Communication Officer-Press relations
02-549 02 78 – 0478 75 01 41

Cathy Verbyst
Communication Officer-Press relations
02-549 02 78 – 0478 75 01 41

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