Project

The project is sponsored
by Parent Project Czech
Republic civil association.

In 2004 work started on the DMD/BMD patients database/registry. The original aim of the database was to effectively integrate Czech patients to international clinical studies. International cooperation and active searching for patients on an international level is necessary because suitable patients matching to the particular clinical and genetic criteria are insufficient in single countries. In the case of finding effective therapeutic methods the central database will speed up selection and indication of appropriate patients.

This registry provides a national database of DMD/BMD patients for medical experts and researchers to help accelerate the development and delivery of new treatments for Duchenne and Becker muscular dystrophy.

The registry of patients with DMB/BMD in the Czech Republic is now available online. This database is available to registered specialist and patients, who have access to their personal data. The registry provides the possibility to collect, view or search for data regarding patients' phenotype and genotype profiles and other medical information for the purpose of future international and intervention studies and to establish a therapeutic strategy. The substance of this project is to collect all the necessary information possible about Czech patients who have this serious hereditary disease.

The registry of Czech and Slovak Duchenne/Becker muscular dystrophy patients is a result of cooperation between the Institute of Biostatistics and Analyses, Masaryk University (technical realization, data analyses), Parent Project Czech Republic civil association (project initiation, financial support) and University Hospital Brno (scientific advisor).

Project goals:

• Describe Czech and Slovak DMD/BMD patients
• Recruit patients for international clinical studies
• Describe prevalence of specific types of mutations in the dystrophin gene
• Speed up selection of appropriate patients in the case of effective treatment
• Encourage research into DMD/BMD
• Improve understanding of DMD/BMD
• Inform participants about new research/latest developments in the area of DMD/BMD treatment
• Publish new results of the research

The patients and data related to them are entered in the registry according to both national and international law (EU directive 95/46/EC, Czech Act No. 101/2000, etc.). The patients records are entered into the database on the basis of an agreement by their legal representative (informed consent).

The aim of the registry is to register every patient with Duchenne/Becker muscular dystrophy in the Czech Republic and Slovakia in a secure, legally protected way. Information obtained in this project will be used for collaboration with worldwideresearch centers for the future development of effective therapy for Duchenne/Becker muscular dystrophy.

Important notes:

• Participation in this project is voluntary
• Participation in this project is free
• The system is secured
• Patients have access to their own data
• The Project respects both national and international laws
• The Project is governed by medical professionals