Imke Ditters receives PhD for research into Pompe disease treatment
3 november 2026
On November 2nd, Imke Ditters received her PhD for her research into the treatment of Pompe disease. Her research provides new insights into enzyme therapy dosing, home-based treatment and treatment effectiveness.
In her thesis, ‘Pushing boundaries in Pompe Disease: On optimal dosing, home-based enzyme replacement therapy, and the impact of anti-drug antibodies', Imke describes more than 25 years of Dutch experience with enzyme replacement therapy (ERT). She investigated how treatment can be further optimized for both children with the classic infantile form and patients with the late-onset form of the disease. Her PhD research was supervised by Professor Ans van der Ploeg, with Dr Hidde Huidekoper and Dr Hannerieke van den Hout as co-supervisors.
One important finding was that children with the classic infantile form who received a higher dose of enzyme replacement therapy (40 mg/kg per week) had better survival rates and were more likely to learn to walk than children receiving the standard dose. These findings have contributed to the increased use of higher-dose treatment in children worldwide.
The research also shows that enzyme replacement therapy can be safely administered at home. This can reduce the burden of frequent hospital visits and contribute to a better quality of life. Imke also investigated the impact of antibodies on treatment. In some patients, high levels of antibodies can reduce the effectiveness of therapy and increase the risk of infusion-related reactions.
These findings contribute to further improving the treatment of Pompe disease. The knowledge gained may also be relevant to other metabolic disorders treated with enzyme replacement therapy.
Congratulations, Imke, on this great achievement!
