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REPORT ON RARE DISEASE RESEARCH, ITS DETERMINANTS IN EUROPE AND THE WAY FORWARD Participation in European projects Bulgaria participates, or has participated, in the following European Reference Networks for rare diseases: Dyscerne and Care-NMD. Bulgaria participates, or has participated, in European rare disease research projects, including: EUROGLYCANET. Bulgaria contributes to the following European registries: EUROCARE CF and TREAT-NMD. Bulgaria contributes to the EUROPLAN project. Discussions at the National Conference on Rare Diseases (28-30 May 2010) highlighted the need to make European rare disease research projects and topics better known amongst researchers and academics in order to set up partnerships. 2.2.2.4. CYPRUS Research activities Funding opportunities for rare disease research are offered by the Cyprus Research Promotion Foundation and the Cyprus Institute of Neurology and Genetics. The Telethon is an international charitable institution which is organised by the Cyprus Institute of Neurology and Genetics (CING) to support scientific research into gene therapy for neuromuscular diseases. A large proportion of net revenue from the Telethon (approximately 30%) is allocated to the Association for Patients with Muscular Dystrophy and the rest supports specific research projects conducted at the Institute. The selection of these investigations is made with the help of an independent international scientific committee. In Cyprus, preparation of the approval of clinical trials using lentivirus vectors for gene therapy in B- Thalassemia is underway. E-Rare Cyprus is currently not a member of E-Rare and does not participate in their calls. Participation in European projects Cyprus participates, or has participated, in the following European Reference Networks for rare diseases: Dyscerne, ENERCA and TAG. Cyprus participates, or has participated, in European rare disease research projects including: EUROPEAN LEUKEMIA NET, Ithanet, LEISHMED and MYELINET. Cyprus contributes to the following European registry: EUROCARE CF. Cyprus contributes to the EUROPLAN project. 2.2.2.5. CZECH REPUBLIC Research activities Rare diseases research is conducted through several funding bodies: the internal grant agency of the Czech Ministry of Health (www.mzcr.cz), the grant agency of the Czech Republic (www.gacr.cz), and the grant agency of the Charles University Prague (www.gauk.cz). Currently, 15 different research projects in the field of rare diseases are registered with Orphanet, focusing on 30 different rare disorders. At least three projects target specific genes. E-Rare The Czech Republic is not currently a partner of the E-Rare research programme on rare diseases. However, negotiations with the E-Rare2 project are underway. Page 18 of 71
REPORT ON RARE DISEASE RESEARCH, ITS DETERMINANTS IN EUROPE AND THE WAY FORWARD Participation in European projects Teams in the Czech Republic participate, or have participated, in the following European Reference Networks for rare diseases: Dyscerne, ECORN CF, EPNET/EPI, ENERCA Paediatric Hodgkin Lymphoma Network, NEUROPED, PAAIR and Care-NMD. Teams in the Czech Republic participate, or have participated, in European rare disease research projects, including: CLINIGENE, ENCE PLAN, EUMITOCOMBAT, EURO-PADNET, EUROCARE-CF, EUROPEAN LEUKEMIA NET, EUROGENTEST, EUROGLYCANET, HUE-MAN, MYORES, NEUROSIS, PNSEURONET, RD PLATFORM, SARS/FLU VACCINE, SCRIN-SILICO and SIOPEN-R-NET. Teams in the Czech Republic contribute to the following European registries: EUROCARE CF, EUROCAT and TREAT-NMD. The Czech Republic is also a partner country of the Severe Chronic Neutropenia International Registry (SCNIR), monitoring the clinical course, treatment, and disease outcomes in patients with severe chronic neutropenia. The Czech Republic also participates in many international-level activities including ERNDIM (a consortium for quality assessment in biochemical genetics for rare diseases) and Europlan, developing guidelines for national rare disease plans. 2.2.2.6. DENMARK Research activities There are no specific programmes for rare disease research in Denmark or focussed calls/grants. Although there are no specific initiatives to support research into rare diseases in Denmark, Danish researchers are active in the field and there are resources in place (biobanks, registries, databases) for rare disease research. E-Rare Denmark is not currently an E-Rare partner. Participation in European projects Danish teams participate, or have participated, in the following European Reference Networks for rare diseases: Dyscerne, EPI, NEUROPED, Paediatric Hodgkin Lymphoma Network, PAAIR, EN-RBD and Care-NMD. Danish teams participate, or have participated, in a number of European research projects for rare diseases, including: ALPHA-MAN, CILMALVAC, EURHAVAC, E-IMD, EMSA-SG, EUROCRAN, EUROGLYCANET, EUROPEAN LEUKEMIA NET, EMVDA, EUNEFRON, HDLOMICS, HUE- MAN, HUMALMAB, LEISHMED, MMR-RELATED CANCER, MYASTAID, NEUROKCNQPATHIES, NEUROPRION, NM4TB, PULMOTENSION, SPASTICMODELS, SIOPEN-R-NET, SERO-TB, TB TREATMENT MARKER and VACCINES4TB. Amongst others, Danish teams contribute to the following European registries: EUROCARE CF, EMHG and EUROCAT. Denmark contributes to the EUROPLAN project and EU Network of experts on newborn screening. 2.2.2.7. ESTONIA Research activities According to the Inventory of Community and Member States’ incentive measures to aid the research, marketing, development and availability of orphan medicinal products, Eesti Teadusfond (Estonian Science Foundation) supports research on rare diseases at the national level on the basis of appropriate applications, but there is no distinction from other projects not related to rare diseases Page 19 of 71
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