Ans T. van der Ploeg MD, PhD is Chair of the Center for Lysosomal and Metabolic Diseases at the Erasmus MC University, Rotterdam, and chair of department for metabolic diseases of Leiden University Medical Center, the Netherlands. The center for lysosomal and metabolic diseases is a joined initiative of the departments of Pediatrics, (Child)Neurology, Internal Medicine, Clinical Genetics and Hospital Pharmacy. It aims to improve treatment, care and diagnosis of children and adults, to stimulate translational research, to provide education and to disseminate information. The center has is a designated rare disease expert center for Pompe disease, Lysosomal storage disorders, hypophosphatasia, urea cycle defects and organic acidurias and Porphyria. It serves as the national reference center for treatment with enzyme replacement therapy of patients with MPS I, MPS II, MPS VI, NCL and Pompe disease. The research performed by the center has an important focus on lysosomal storage disorders and in particular Pompe disease, and includes clinical research (long term effects of ERT, dosing, immunomodulation, patient reported outcome) as well as development of innovative therapies (Gene, RNA based and cell based therapies) and implementation of next generation (enzyme) therapies. The center collaborates since 2002 with the International Pompe Association on the IPA survey. The Center is governor of the international Pompe mutation database  Van der Ploeg is vice-coordinator of the European Pompe Consortium (EPOC), metabERN and coordinator of the LSD subnetwork of metabERN, and member of EuroNMD (European reference network for Neuromuscular Disorders). She is member of several scientific advisory boards, member of the council of the Society of the Study Group of Inborn Errors of Metabolism (SSIEM). She was Chair and organizer of the SSIEM symposium named “building bridges” held in the Doelen in Rotterdam in 2019, which was attended by more than 3000 participants from 86 countries. The symposium focused in  particular on innovative therapies such as gene therapy, regenerative medicine, RNA based therapies and potential benefits from other fields.
Van der Ploeg received her MD “with honors” in 1985 at the Erasmus University. From 1985 till 1989 she worked at the Department of Cell Biology and Clinical Genetics on the feasibility of enzyme replacement therapy in cellular models for Pompe disease. Since then she has been involved in the multiple steps leading to the development of enzyme replacement  therapy: Cloning of the gene, biotechnological production of recombinant human alpha-glucosidase (the enzyme deficient in Pompe disease) in milk of transgenic mice and rabbits, and in CHO cells; development of a knock-out model for Pompe disease; feasibility studies in mice and finally the first clinical trial in infants; and later the international multicenter placebo controlled trial that showed effects of therapy in adults. The current work of the center on implementation and development of new therapies builds on this experience. Van der Ploeg received several awards for her work such as “The Research award for young investigators” from Erasmus University Rotterdam, the “Pharming therapy award”, and “het kroontje” from the Prinses Beatrix Spierfonds. She has published over 200 publications in peer reviewed international journals and books.