EJP RD training courses services

Provided by European Joint Programme on Rare Diseases (EJP RD)

EJPRD

The EJP RD Training and Empowerment program aims to develop knowledge and build capacity of the rare disease (RD) research and care community through the delivery of training programs on a wide range of relevant topics thereby providing opportunities to a large panel of stakeholders, including:
* DATA MANAGEMENT & QUALITY TRAININGS
The main objectives of the training activities on data management & quality are to decrease rare disease data fragmentation and increase data quality by raising the level of capacities and help data sharing in a FAIR ecosystem. The training activities cover a variety of topics including but not limited to variant interpretation, data management, registries, FAIRfication processes, Orphacodes, biobanking, and undiagnosed cases.
*PATIENTS & RESEARCHERS TRAININGS
The overall objective of this training module is to improve rare disease research and innovation and to enhance the uptake of research results by building the capacity of the patient community and other key stakeholders. Moreover, dedicated educational activities will be aimed to train “expert” paediatric patients on rare diseases, paediatric medicines development and clinical research.

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EUNetHTA health technology assessment services

Provided by European Network for Health Technology Assessment (EUNetHTA)

EUnetHTA develops several services that help inform the decision-making process around the introduction of pharmaceuticals and other technologies at the national, regional and European levels. These include Joint assessments, early diologues and Post Launch Evidence Generation (PLEG)
* Assessment submission: Health technology companies are encouraged to contact us via the following two channels if interested in a EUnetHTA assessment of their product. These are distinguished by EUnetHTA as Pharmaceuticals and Other Technologies (i.e. non-pharmaceuticals).
*Early dialogue: parallel scientific advice with the European Medicines Agency. This aims to allow medicine developers to obtain feedback from regulators and health technology assessment (HTA) bodies on their evidence-generation plans to support decision-making on marketing authorisation and reimbursement of new medicines at the same time.
* Horizon Scanning: topic identificatin, selection and Prioritisation for HTA cooperation

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EUPATI Training courses services

Provided by European Patients' Academy (EUPATI) toolbox

EUPATI provides training for patients and patient representatives on the end to end process of medicines research and development. Trained patient experts are the core resource for patient involvement in medicines R&D, regulatory deliberations, and other patient engagement initiatives. EUPATI is committed to changing the face of patient engagement through patient education.

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EJP RD patient empowerment and engagement services

Provided by European Joint Programme on Rare Diseases (EJP RD)

EJPRD

The EJP RD has developed a short guide on patient partnerships in rare diseases research projects. It aims to encourage fruitful, sustainable and enduring partnerships between scientists and patient organisations, co-leading the way for systematic patient-centered research.

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MD-NET disease/ translational resource services

Provided by MD-NET

Guidelines and advice for professionals and patients
Best Practice Guidelines on molecular diagnostics in Duchenne / Becker muscular dystrophies (2010)
Diagnosis and management of Duchenne muscular dystrophy
Consensus statement for standard of care in spinal muscular atrophy (2007)
Heart failure DEGAM guideline No. 9 (2006)
Diagnostics and therapeutic approaches for mitochondrial disease in children and adolescents – S2 guideline (2009)
Diagnosis of myopathies (2008)
Guidelines for the molecular genetic diagnosis of muscular dystrophies Duchenne and Becker (1999)
Consensus statement on standard of care for congenital muscular dystrophies (2010)

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ID-EPTRI ethical / legal issues, informed consent services

Provided by ID-EPTRI

EPTRI

EPTRI provides centralised services provided by EPTRI experts of the Central Hub and partner Institutions covering regulatory, ethical and exploitation users’ needs.

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ID-EPTRI development of project towards a medical application services

Provided by ID-EPTRI

EPTRI

EPTRI provides integrated services provided by the Thematic Research Platforms in EPTRI in the following fields:
1. Paediatric Medicines Discovery
2. Paediatric Biomarkers and Biosamples
3. Developmental Pharmacology
4. Paediatric Medicines Formulations

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PedCRIN multinational clinical trial (establishment / management / monitoring) services

Provided by PedCRIN

PedCRIN developed a series of tools (WP3) and procedures to support the setup and the management of multinational neonatal and paediatric clinical trials in Europe. These tools are developed to train and support researchers and clinicians so they can establish, run, and manage both paediatric and neonatal multinational clinical trials in a more effective and easier way. The removal of barriers to trial management will speed up the evaluation of new and improved therapies.

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SYSBIO – Centre of Systems Biology systems biology expertise services

Provided by SYSBIO – Centre of Systems Biology

SYSBIO

M4 – MAKE ME MY MODEL consultancy service – Flux Balance Analysis
M4 – Make Me My Model is an initiative of ISBE light, the starting pilot of ISBE. At SYSBIO, you can contact us to develop a metabolic math model specific for your research. This constraint-based model approach, called Flux Balance Analysis, could be of great usefulness to understand your data and improve the impact of your publication (i.e. better journal, wider and multidisciplinary approach, etc.). Different approaches can be provided, thanks to ISBE partners.

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ISBE NL systems biology expertise services

Provided by ISBE NL

Make Me My Model ( 4M 4U): Non modellers can obtain (assistance with) the making of a computational model of their biological system. If we can, we shall make you your model such that you can run it, use it to make predictions of the schemes you have in mind and compare that with your experimental results. Contact isbe@nl to make you a model of an experimental biological or medical system you are researching. In this way we can establish whether we have the modelling expertise to help you or can refer you to other isbe nodes

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