The ERICA Patient Reported Outcome Measures (PROMs) Repository

The ERICA Patient Reported Outcome Measures (PROMs) Repository is the first attempt to identify and centralize Clinical Assessment Outcomes questionnaires of relevance for rare diseases and constitutes a milestone in the Europe-wide standardization of Patient-Centered Outcome Measures (PCOMs) and PROMs for rare diseases. It has been made possible through the joint collaboration between Orphanet, Mapi Research Trust/ICON and ERN EuroBloodNet (VHIR, APHP), and the active contribution of ERNs and ePAGs. From Endo-ERN prof. Nienke Biermasz (LUMC) have participated in the ERICA WP3 PCEWG (Patient-centred expert working group) and contributed in this first version. Via ERICA you [...]

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ERICA Educational Webinar 2 “Practical guide on how to use the Catalogue of services and the IMT”

In this ERICA webinar, Anton Ussi (Operations & Finance Director at EATRIS), together with Agustin Arasanz Duque (Senior Innovation Manager at EATRIS), will provide practical guidance for using two important tools for RD researchers, developed within EU-funded projects: ERICA and EJP RD. This webinar is part of the Educational Webinar Series from ERICA’s WP5 Translation and Innovation. It builds on the introductory webinar “Current research services available for the rare diseases community” that was organized in November 2021 (available on the ERICA website Continue reading

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IRDiRC Announces The Creation of Regulatory Science Committee to Tackle Regulatory Challenges in Rare Disease Research

IRDiRC, a global collaborative initiative with the vision to enable all people living with a rare disease to receive an accurate diagnosis, care, and available therapy within one year of coming to medical attention, today announced the formation of the Regulatory Science Committee (RSC). The new RSC “brings broad expertise across different stakeholders and geographies allowing for a balanced synergy that will provide insights and guidance into the advancement of IRDiRCs mission” said David A. Pearce, Chair of IRDiRC.

Read the full press release here: https://irdirc.org/irdirc-announces-creation-of-regulatory-science-committee/

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ERN Research Training Workshops funding opportunity

The ERN Research Training Workshops funding opportunity is now open for applications until October 15th, 2022. The goal of the workshops is to train researchers and clinicians affiliated to ERN- Full  Members or – Affiliated Partners in relevant topics on research in rare diseases. Training themes may include innovative research methodologies, diagnostic research topics, interdisciplinary treatment approaches, such as gene therapy and transplantation, etc. Moreover, the workshops will be aiming to provide a cross-ERN added value.

The workshops will be delivered as two-day events. The costs for the workshop organization will be covered up to a limit of €25,000 (venue, [...]

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Last round for the Research Mobility Fellowships funding opportunity

The European Joint Programme on Rare Diseases (EJP RD) is glad to announce that the last round for the Research Mobility Fellowships funding opportunity will open on October, 3rd, 2022. The call aims to support PhD students, postdocs, and medical doctors in training to undertake scientific visits fostering specialist research training outside their countries of residence.

The exchange can be carried out (1) within the same ERN (Full Members and Affiliated Partners), (2) between different ERNs (Full Members and Affiliated Partners), or (3) between ERN Full Members / Affiliated Partners and non-ERN institutions.

Either home or host (secondment) institution must [...]

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Mini-survey of the Dutch Pituitary Foundation

The Dutch Pituitary Foundation, the Wilhelmina Children’s Hospital and the Princess Máxima Center in Utrecht in collaboration with European Reference Network for Rare Endocrine Conditions (Endo-ERN) are collecting the opinions and experiences of patients (and/or carers) living with the consequences of hypothalamic dysfunction, or problems in the pituitary/hypothalamus area after treatment for a tumour in the hypothalamic/pituitary region.

We would like to know what patients consider important about their condition and which topics they believe should be given priority in healthcare, in research, and on how to function in everyday life.

We are looking for patients who have been diagnosed [...]

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Meet our new ePAG Diana Kwast-Hoekstra

Diana Kwast

I would like to introduce myself to you as a new member of the Endo-ERN ePAG MTG1, from the end of May 2022. I have been nominated by the Dutch Adrenal Society NVACP and from the 25th of May I will take over from Mr. Johan Beun, whom does intend to retire in the summer of 2022.

My connection with adrenal disorders is that I myself since 2016, have been suffering from steroid-induced adrenal insufficiency and diabetes due to prednisone use, in severe asthma.

In my search for information about this adrenal disorder and its treatment, I came into contact [...]

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GloBE-Reg: Global Registry For Novel Therapies In Rare Bone & Endocrine Conditions

GloBE-Reg is a new international registry project that has been launched by the Office for Rare Conditions at the University of Glasgow. The project believes that post regulatory approval studies may be more effectively performed through registry-based studies that collect relevant real world data through a joint collaborative approach between scientific centres, industry partners and medical societies. GloBE-Reg will also use this approach to serve the needs of the endocrine community for assessing longer term clinical outcomes, which are particularly important to continuously expand our scientific knowledge and improve care in children.

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