Should you participate in a clinical trial?

The doctors who are now treating you are probably the best people to talk to for answering this question. They are obligated to give full information about the trial and also explain the possibilities for treatment options outside of the trial.

To help you in your personal decision for or against participation, we offer you a downloadable brochure. The brochure answers most of the questions you might have about the trials. It explains what studies are, how they are planned and conducted. It describes the opportunities and risks generally associated with participation. It tells you what your rights as a participant are and what the doctors who are supervising the trial are obligated to do.

More information about clinical studies, offers "vfa. - Die forschenden Pharmaunternehmen" in its brochure. (Brochure only avaiable in german language).

An overview of the current therapy concepts with gene therapy, haematopoietic stem cell transplantation (bone marrow transplantation), enzyme replacement therapy, substrate reduction therapy and chaperone therapy is scientifically presented in the following article:

Pinto, E. Vairo F., D. Rojas Malaga, F. Kubaski, C. Fischinger Moura de Souza, F. de Oliveira Poswar, G. Baldo, and R. Giugliani. "Precision Medicine for Lysosomal Disorders" Biomolecules 10, no. 8 https://dx.doi.org/10.3390/biom10081110 (Jul 26 2020).

We have illustrated the topic of therapeutic concepts for lysosomal diseases in a comic strip in which we try to explain what is not working in the cell and, more importantly, how it could be fixed.