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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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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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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The Endo-ERN newsletter November 2020 is out now!
Read more about the upcoming & recording Endo-ERN webinars, New MTG8 chairs, Launch of Exchange Programme for healthcare professionals in European Reference Networks, The ERICA Project and much more.
The newsletter can be found here.
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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 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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The Alstrom Syndrome Global online Conference to be held on the 4th and 5th December.
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On Friday December 18th at 13:00 – 15:00 CET an Endo-ERN webinar is organised with multiple speakers within the main thematic group of “Sex Development & Maturation”
Program:
13:00 Introduction by the chairs Olaf Hiort, Luca Persani, Manuela Brosamle
& Arlene Smyth
13:10-13:20 Olaf Hiort, Lubeck, Germany
German DSD network and quality of care
13:20-13:40 Sasha Howard, Queen Mary University of London, UK
Genetics of delayed puberty: differential architecture of CDGP vs CHH
13:40-13:55 Martine Cools, University of Gent, Belgium
Management of GD in Europe: an update in pediatric care
13:55-14:10 Guy T’Sjoen, [...]
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Glasgow Webinar Series in Rare Conditions: Hypophosphatasia – Diagnosis and Management
Tuesday 8th December 17.00 -18.30 (GMT) 18.00-19.30 (CET)
17:00 Introduction – S. Faisal Ahmed, Glasgow
17:05 A case of infantile HPP – James Irvine, Glasgow
17:15 The aetiology and clinical presentation of HPP – Agnès Linglart, Paris
17:40 The management of HPP across the life span – Anya Rothenbuller, Paris
18:15 Q&A
18.30 Close
The Glasgow Webinar Series is supported by the Glasgow Children’s Hospital Charity and An unrestricted education grant from Alexion.
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On Thursday December 3rd at 17:00 – 18:00 CET an Endo-ERN CMPS webinar about “How to enrol a patient, start a panel and upload files to a panel is organised”
During this webinar you will learn how to create a CPMS panel. We will show you how to enrol a patient, how to start a patient panel and how to upload files to your panel.
After the presentation our operational helpdesk members (Isabel Moenig and Danielle Steenvoorden) will be available to answer your questions during the live Q&A.
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