Fourth Eastern European Conference for Rare Diseases and Orphan Drugs “Together for Integrative Approach to Rare Diseases” 13-14 June 2009 - Plovdiv, Bulgaria In collaboration with neuromuscular disease neuromuscular disease Ysbrand Poortman Founder/ board member VSN, Dutch Association for NeuroMuscular Diseases EAMDA, Eur. Alliance Neuromuscular Disease Associations ENMC, Eur. NeuroMuscular Center for stim. & coord. of research EPPOSI,Eur. Platform Patient Organisations, Science & Industry Vice president WANDA, World Alliance Neuromuscular Disorder Associations
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Fourth Eastern European Conference for Rare Diseases and Orphan Drugs “Together for Integrative Approach to Rare Diseases”
Fourth Eastern European Conference for Rare Diseases and Orphan Drugs “Together for Integrative Approach to Rare Diseases”
13-14 June 2009 - Plovdiv, Bulgaria
In collaboration with
neuromuscular diseaseneuromuscular diseasecontent:characteristicsimpact of diseasehealthcare services
models counteractions - disability manus robot arm- social field AFM-telethon- medico scientific field
european neuromuscular center (ENMC) “Patients’ Roadmap to Treatment”
contributions to medicine developmentnear future
Fourth Eastern European Conference for Rare Diseases and Orphan Drugs “Together for Integrative Approach to Rare Diseases”
13-14 June 2009 - Plovdiv, Bulgaria
In collaboration with
neuromuscular diseaseneuromuscular disease
Main characteristics
• paralysing due to mal functioning of cellsin spinal cord, muscle cells or conjunction
• progressive, often life-threatening• most are genetic (all patterns) and rare•no cures (?), • complex management to counteract implications of disease
Fourth Eastern European Conference for Rare Diseases and Orphan Drugs “Together for Integrative Approach to Rare Diseases”
13-14 June 2009 - Plovdiv, Bulgaria
In collaboration with
ENMC ORGANIZATIONENMC ORGANIZATION
EXECUTIVE COMMITTEEEXECUTIVE COMMITTEEPatient organizations (8 voting members)Patient organizations (8 voting members)in charge of management, policy, promotion, in charge of management, policy, promotion, implementation of outcomeimplementation of outcome
RESEARCH COMMITTEERESEARCH COMMITTEE11 elected members11 elected membersin charge of all sciencein charge of all science
Fourth Eastern European Conference for Rare Diseases and Orphan Drugs “Together for Integrative Approach to Rare Diseases”
13-14 June 2009 - Plovdiv, Bulgaria
In collaboration with
Professional backgrounds Workshops participants 2004
Paediatrics16%
Others11%
Neurology33%
Basic sciences27%
Pathology4%
Genetics9%
Fourth Eastern European Conference for Rare Diseases and Orphan Drugs “Together for Integrative Approach to Rare Diseases”
13-14 June 2009 - Plovdiv, Bulgaria
In collaboration with
DISSEMINATION OF KNOWLEDGEDISSEMINATION OF KNOWLEDGEbooks, workshop reports, scientific papers, website, presentation and posters at international meetings
Fourth Eastern European Conference for Rare Diseases and Orphan Drugs “Together for Integrative Approach to Rare Diseases”
13-14 June 2009 - Plovdiv, Bulgaria
In collaboration with
Beyond presentations…Beyond presentations…
…much interaction, interactivity,
and socialization…
…leading to more collaboration
Fourth Eastern European Conference for Rare Diseases and Orphan Drugs “Together for Integrative Approach to Rare Diseases”
13-14 June 2009 - Plovdiv, Bulgaria
In collaboration with
A few group picturesA few group pictures
Minocycline in ALS Inducing Hypertrophy
Fourth Eastern European Conference for Rare Diseases and Orphan Drugs “Together for Integrative Approach to Rare Diseases”
Better Better networkingnetworking with other centres with other centres Muscular Dystrophy Association (USA)Muscular Dystrophy Association (USA) Asian Oceanian Myology CenterAsian Oceanian Myology Center
Integration of Eastern European countriesIntegration of Eastern European countries and developing/emerging countriesand developing/emerging countries
Secure funding (including thrSecure funding (including throughough the E.U.) the E.U.)
European NeuroMuscular Center
Fourth Eastern European Conference for Rare Diseases and Orphan Drugs “Together for Integrative Approach to Rare Diseases”
13-14 June 2009 - Plovdiv, Bulgaria
In collaboration with
WANDAWANDA
“seeks a world in which neuromuscular disorders are understood and eradicated and in which the people affected are well supported”.
Quadrennial assembly, 50 national NMD-ass.Support emerging/new associations
Fourth Eastern European Conference for Rare Diseases and Orphan Drugs “Together for Integrative Approach to Rare Diseases”
13-14 June 2009 - Plovdiv, Bulgaria
In collaboration with
Objective 4:Objective 4:
“to facilitate swift dissemination of information on treatment and research findings”
Warnings for fake treatments:
myoblast transfer, stemcell therapy in China
Fourth Eastern European Conference for Rare Diseases and Orphan Drugs “Together for Integrative Approach to Rare Diseases”
13-14 June 2009 - Plovdiv, Bulgaria
In collaboration with
Sharing Best PracticesSharing Best Practicesneuromuscular diseaseneuromuscular disease• WANDA• Istanbul 2006• Patients roadmap to treatment
Fourth Eastern European Conference for Rare Diseases and Orphan Drugs “Together for Integrative Approach to Rare Diseases”
13-14 June 2009 - Plovdiv, Bulgaria
In collaboration with
Pathways leading to therapy (1986) Pathways leading to therapy (1986) (2006)(2006)