Endo-ERN CPMS webinar: How to invite members to your panel, schedule a video meeting and join a video meeting

On Thursday January 28th at 17:00 – 17:30 CET an Endo-ERN CPMS webinar about “How to invite members to your panel, schedule a video meeting and join a video meeting“ is organised.

Danielle Steenvoorden from the Operational Helpdesk will give you an overview of the following steps after opening a patient panel within CPMS: how invite members and schedule and join a video meeting. At the end of this webinar there will be a live Q&A for all your questions.

This is the second part of a series of webinars about CPMS.

The first webinar “CPMS: how to enroll a [...]

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Rare Disease Day

Rare Disease Day takes place on the last day of February each year. The main objective of Rare Disease Day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients‘ lives.

The campaign targets primarily the general public and also seeks to raise awareness amongst policy makers, public authorities, industry representatives, researchers, health professionals and anyone who has a genuine interest in rare diseases.

Building awareness of rare diseases is so important because 1 in 20 people will live with a rare disease at some point in their life. Despite this, there [...]

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Research Training Workshops for ERN members

In September 2020 the EJP RD launched its second call of the “European Reference Networks (ERNs) Training and support program” as part of the ‘Training and Empowerment’ activities that aim to fill the gap in education on rare diseases research.

The “Research Training Workshop” call is aimed at identifying workshop topics to train ERN researchers and clinicians in relevant innovative themes with a cross-ERN added value. Selected applicants will receive financial support for the organization of a 2-days workshop for 20 participants.

Training themes include innovative research methodologies, diagnostic research methodologies, interdisciplinary treatment approaches, such as gene therapy [...]

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EJPRD internal call for innovation projects in clinical trials methodologies information/network meeting

In the context of the EJP RD internal call for innovation projects in clinical trials methodologies in limited populations, that opened on 7th of December 2020 and will close on 3rd of March 2021, an “Information/Networking Event” will be organized on the 25th of January 2021. The meeting will take place online from 15:00 to 18:30.

All interested applicants are invited to participate in this event. The objectives of the meeting are as follows:

Presentation of the call modalities and objectives followed by Q&A session with participants Presentation of various perspectives of the remaining challenges that the present call will [...]

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EJPRD’s Internal call for innovation project in clinical trials methodology

The Internal Call for Innovation Project in Clinical Trials Methodology in Limited Populations has been launched on December 7, 2020. The call is open to EJP RD beneficiaries and their linked third parties.

The innovation methodologies topics particularly include (but are not limited to):
· Development of a disease progression model from a natural history cohort or other observational studies.

· Development and validation of a disease specific clinically meaningful outcome with special interest in PCOMs, or composite endpoints.

· Development of a design and analysis procedure for a pharmacometric model and/or bridging study.

· Development of a randomization-based [...]

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Endo-ERN webinar: Endocrine and bone management in Duchenne muscular dystrophy

In this webinar, Dr. Jarod Wong from the University of Glasgow will review the endocrine and bone morbidity in DMD with focus on the updated international care recommendations and discuss areas for future research which includes management of endocrine and bone in adults with DMD. He will be joined by Justus Kuijer who will share his experience of endocrine and bone morbidity.

Duchenne muscular dystrophy (DMD) is a rare X-linked inherited neuromuscular condition affecting 1 in 4000 boys. Most are diagnosed in early childhood, and it is characterised by progressive muscle wasting. To date, there is no curative therapy, and [...]

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Launch of Exchange Programme for healthcare professionals in European Reference Networks

Knowledge sharing and stimulating collaboration between health care professionals in European Reference Networks (ERN). That is the aim of the Exchange Programme 2020-2022, funded by the European Commission. In the coming two years, three different rounds of exchanges will take place between professionals of HealthCare Providers that are member of an ERN. The first round of visits will start in March 2021.

The objective of the ERN Exchange Programme is to palliate disparities in specific knowledge or gaps in expertise by facilitating the arrival of high-level expertise in a considerable number of diseases to a big number of Healthcare Providers.

 [...]

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The ERICA Project: joining forces to integrate research and innovation capacity across all 24 European Reference Networks

The European Rare disease research Coordination and support Action (ERICA) Project received a positive evaluation for a H2020 grant of 2.3 million to establish a structural framework in support of the research activities of the ERNs.

ERICA will strengthen research and innovation capacity by integration of ERN research activities, outreach to European research infrastructures to synergistically increase impact and Innovation. This will result in safe, accessible and efficient access of therapies for the benefit of patients suffering from rare diseases and Conditions.

Rare diseases

Rare diseases are defined as diseases that affect not more than 1 person per 2000 in [...]

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