16th International Postgraduate Course on Lysosomal Storage Disorders took place on 7th-10th of June, 2017 in Nierstein, Germany. Course was attended by a physician from Coordinating Centre for Children’s Rare Diseases, Viktoras Sutkus.
Lectures in the course were read by famous lecturers, specialists of rare lysosomal diseases – Michael Beck, Roberto Giugliani, Dominique Germain, Konrad Sandhoff and others, as well as MPS Society patient representative Sophie Thomas.
During the course, biology and pathophysiology of lysosomes, genetics, diagnostics and treatment of rare disases, patient role in the diseases treatment were discussed. More attention was given to Fabry, Gaucher, Pompe diseaes, mucopolysaccharidoses, leukodystrophies. Physicians have given lectures on orthopedic management, eye damage, neurodegenerative mechanisms in lysosomal storage diseases. New treatment methods were also discussed – enzyme replacement therapy news, blood stem cell transplantation, chaperone therapy, gene therapy, tools to transfer drugs through the blood-brain barrier. After lectures and during breaks, course participants had a chance to talk to experienced physicians about rare diseases, receive more information on lysosomal storage diseases or discuss treated patients and difficult cases. The course was attended by 18 physicians, scientists and pharmaceutical specialists from all over the world.