Professor Frances Platt, Ph.D.
Scientific Advisor through Oxford University Consulting
- Discovered and was instrumental in developing substrate reduction therapy for Lysosomal Storage Diseases (Zavesca, Actelion)
- Internationally recognised expert in lysosomal storage diseases with more than 125 publications in peer reviewed journals including Nature, Science, Nature Genetics, and Nature Medicine
- Professor of Biochemistry and Pharmacology in the Department of Pharmacology, University of Oxford, UK
Christine í Dali, MD
Scientific Advisor
- Consultant Pediatrician, Department of Clinical Genetics, Copenhagen University Hospital.
- Principal investigator on the clinical trials for enzyme replacement therapy in the lysosomal storage disease metachromatic leukodystrophy (MLD).
- Partner and investigator of the EU driven alpha-mannosidase project.
- Member of the advisory board of the focus group for orphan drugs in Denmark.