VUB researchers prof. dr. Thierry VandenDriessche and prof. dr. Marinee Chuah received €50.000 from the Duchenne Parent Project Belgium, a non-profit of parents and family members of children with Duchenne muscular dystrophy. With this collaboration, the non-profit wants to support the preclinical gene therapy program for Duchenne muscular dystrophy of the researchers.
DMD (Duchenne Muscular Dystrophy) is a degenerative, hereditary muscle disorder that affects about 1 in 5,000 boys. DMD is caused by a genetic defect in the dystrophin gene. Patients with DMD typically suffer from progressive muscle weakness, heart and respiratory failure. Even with the best available treatments that are currently available, the average life expectancy is only 30 years. It is a genetic disorder of high unmet medical need.
The field of gene therapy is gaining momentum consistent with successes achieved in clinical trials for many hereditary diseases. The VUB team headed by prof. dr. Thierry VandenDriessche and prof. dr. Marinee Chuah has been conducting research in gene therapy for DMD for more than 10 years now. The support of DPP Belgium frames in the context of World Duchenne Awareness Day on September 7th: a day dedicated to raise awareness for those affected by Duchenne Muscular Dystrophy.
From left to right: prof. dr. Chuah and prof. dr. VandenDriessche
Prof. dr. VandenDriessche explains why the funding by the Duchenne Parents Project is important: “We feel humbled to receive financial support from the Duchenne Parent Project Belgium and are very grateful to the patients and their families and friends for their trust and support. This funding will certainly help us towards developing the next-generation gene therapy for DMD patients”.
Prof. dr. Chuah is equally enthusiastic: “Our goal and ambition is to achieve long-term therapeutic effects, possibly even a real cure, by gene therapy using our technology that we developed and that may potentially be more efficacious and safer than the state of the art”.
The funding will be used to support the preclinical research program to first test the efficacy and safety of innovative gene therapy technologies in DMD animal models that mimic the cognate human disease.
Read more about the Duchenne Parent Project Belgium at: www.duchenneparentproject.be
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