Preceptorship programme on rare diseases: Mucopolysaccharidosis, management and treatment

The Centre for Rare Diseases at the Helios Horst Schmidt Kliniken (HSK) in Wiesbaden, in collaboration with the Brains for Brain Foundation and as part of the EU-funded project InNerMeD-I-Network, is greatly committed not only to the management of patients but also in activities aimed to increase as much as possible the awareness and the training of medical professionals on neurometabolic diseases, with a particular effort on Lysosomal Diseases. To this aim, in collaboration with Genzyme and SHIRE, are being organised 2 Courses taking place at the Center for Rare Diseases at the Children’s Hospital of the HSK in Wiesbaden from November 2nd to 4th and from November 30th to December 2nd,  2015, respectively. Each course will guest 10 physicians from different countries.

 Objective – The overall objective of the 2 courses is to train professionals with a scientific background and an interest on Lysosomal Storage Diseases, in particular Mucopolysaccharidoses (MPSs) and to provide them with theoretical expert knowledge and practical experience in both the recognition of these diseases and related management. The practical experience in management and treatment of MPSs will be gained through the use of patient case studies that the participants will work on throughout the course and in parallel with new topics being added to their theoretical knowledge base.

Expected results – The main expected results of this program consist in ensuring safe and effective initiation of therapies in patients from countries where there is no experience of administration; decreasing the possibility of severe adverse events during treatment and helping patients to receive a state of the art follow-up.

Learning outcomes – At the end of the course, participants will be able to discuss a multidisciplinary approach for the management and treatment of these complex disorders assuring an adequate follow-up of patients by evaluating the status of the disease.

Here you can find a draft of the almost defined programme indicating the scheduled sessions.