Screen4Rare and MEP Alliance for Rare Disease Newborn Screening

Screen4Rare and its founding organisations (IPOPI, ISNS,  and ESID) are pleased to share the meeting report from the recent Screen4Rare  hybrid event on June 29, in Brussels, organised with the support of the MEP Alliance for Rare Disease Newborn Screening (NBS).

The event, which was held in connection with International Neonatal Screening Day, focused on “Establishing Best Practices – How Can Newborn Screening Be Supported in the European Union.” It was hosted by the Member of the European Parliament (MEP) Stelios Kympouropoulos (EPP, Greece), and moderated by Mr Johan Prévot, Executive Director of the International Patient Organisation for Primary Immunodeficiencies (IPOPI).

This first event by Screen4Rare and the MEP Alliance for Newborn Screening for Rare Diseases received significant interest from Key Opinion Leaders around the world, with over 100 participants in-person and online.

The next event will take place during the second half of 2022 and the date will be available in IPOPI’s calendar.

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AGORA: New consortium for improved gene therapy access in Europe

IPOPI was pleased to represent the PID patients’ community at the first meeting of the AGORA (Access to Gene therapies fOr RAre disease) consortium. The new consortium, launched on September 22, aims to facilitate access to gene therapies for patients with ultra-rare diseases, in Europe. The meeting was held at London’s Great Ormond Street Hospital for Children (GOSH) and attended by founding members from six countries representing academic groups, regulators, funders, patient advocacy groups and drug developers.

Long and short-term objectives

Ultimately, AGORA aims to create an independent, sustainable, not-for-profit entity that can support marketing authorisation, delivery and access to therapies which are not commercially sustainable and therefore would be unavailable to patients.

In the short-term, AGORA will seek to explore the hurdles that currently exist and identify areas of potential change to enable sustainable access to treatments, highlighting better the scientific value, competence and high expertise of the consortium which will provide new ideas and solutions. IPOPI is committed to continuing its collaboration with the AGORA consortium and all relevant stakeholders in the field of gene therapies.

Read the AGORA press release.

 

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