PARENT PROJECT CZECH REPUBLIC

THE PARENT PROJECT CZECH REPUBLIC Patient Nonprofit Organization of DMD/BMD and its model of the involvement in the Research and Drug Development proccess 1. Foundation establishment and goals determination 2. The Parent Project and The FN Pediatric Neurology Clinic (Teaching Hospital) in Brno – Joint Projects 3. Project of DMD/BMD Patients Database 4. Resources Financial 5. Next Steps PP CR

1. Foundation Establishment and Goals Determination The civic association Parent Project has been worked in the Czech Republic since Its aim is to support research and prospective cure of patients suffering from DMD/BMD. One of the conditions for achieving this objective was to centralize the DMD/BMD patients and to gain neurologists eventually geneticists who would be ready to co-operate on common projects in the area of diagnostics and research.

Parents and children meeting – Orlík 2005

Creativ Centre in Smržovka 2006

2. The Parent Project and The FN Pediatric Neurology Clinic (Teaching Hospital) in Brno – joint projects The key moment was joining of MUDr. Peter Vondráček from the TH, Pediatric Neurology Clinic in Brno (TH PNC Brno) to the Parent Project activities result of what was the first contract between the Parent Project association and the TH PNC Brno. Parent Project provided significant financial support to the TH in Brno and also to the Masaryk University which has been determined for diagnostic device and genetic laboratory equipment. In the year 2004, the PARENT PROJECT association granted a significant donation to the Centre for Diagnosis and Therapy of Muscular Disorders in Brno. The Centre provides a multidisciplinary collaboration of specialized departments centred on a complex treatment of patients with muscular dystrophy. Such medical care is not offered in any other place in the Czech Republic.

A large amount of supplies that allows the top-quality genetic testing, which can specifically identify genetic mutations causing Duchenne or Becker Muscle Dystrophy (DMD/BMD) was purchased by the Parent Project Association for the Centre of Molecular Biology and Gene Therapy. In addition, laboratory probes were acquired for the Institute of Pathology and Anatomy. The probes are indispensable for analysis of muscle biopsies and for diagnostics of patients and potential carriers. Furthermore, equipment for the neurophysiology laboratory of the Institute of Pediatric Neurology was purchased, particularly specialized electromyographic needles, which significantly improved muscle examination as well as lessens trauma of myopathic patients. A large amount of supplies that allows the top-quality genetic testing, which can specifically identify genetic mutations causing Duchenne or Becker Muscle Dystrophy (DMD/BMD) was purchased by the Parent Project Association for the Centre of Molecular Biology and Gene Therapy. In addition, laboratory probes were acquired for the Institute of Pathology and Anatomy. The probes are indispensable for analysis of muscle biopsies and for diagnostics of patients and potential carriers. Furthermore, equipment for the neurophysiology laboratory of the Institute of Pediatric Neurology was purchased, particularly specialized electromyographic needles, which significantly improved muscle examination as well as lessens trauma of myopathic patients.

In 2004 started the work on DMD/BMD patients` database creation in cooperation with the Masaryk University in Brno which has been financially supported by Parent Project association. The aim of the database was to integrate Czech patients to foreign clinical studies. International cooperation and active searching for patients on the international level is necessary because suitable patients matching to the particular clinical and genetic criteria are insufficient in the single countries. In case of finding effective therapeutic methods the central database will speed up selection and indication of appropriate patients. The database involves also patients from Slovakia. In 2004 started the work on DMD/BMD patients` database creation in cooperation with the Masaryk University in Brno which has been financially supported by Parent Project association. The aim of the database was to integrate Czech patients to foreign clinical studies. International cooperation and active searching for patients on the international level is necessary because suitable patients matching to the particular clinical and genetic criteria are insufficient in the single countries. In case of finding effective therapeutic methods the central database will speed up selection and indication of appropriate patients. The database involves also patients from Slovakia. The Database is funded exclusively from the resources of the Parent Project. 3. Project of DMD/BMD Patients Database

4. Resources Financial 4. Resources Financial The Parent Project has resources partly from private donors, partly from its own advertising and charitable activities. After five years working stands the Parent Project again in front of a key moment. This time is it to make its work professional. The Parent Project has resources partly from private donors, partly from its own advertising and charitable activities. After five years working stands the Parent Project again in front of a key moment. This time is it to make its work professional. Lukáš Bauer - Olympic medallist WOG Torino 2005

Next Steps of PP in Czech Republic In the next future we would like to support directly research activities either in the Czech Republic or abroad. This great project can by scarcely funded from own resources. It is necessary to gain grants and subsidies from government and EU. Parents in the Czech Republic would like to sponsor the research of the mode called „Exon skipping“. The most successful project of our activity is launching DMD/BMD patients database and diagnose enhancement. Other significant activity is information system and integration of the patients to their healthy fellows. The Parent Project is a partner not only for parents but also for neurologists and geneticists. It is an association which is small as for its number participants but big as for its mission. Our aim is joining to particular research DMD/BMD project. The Parent Project is small organization with great mission.