Pre and Post-Conference Events

In anticipation of the 2024 ECRD conference, an exciting series of podcasts, thought leader sessions, and satellite meetings will be offered to spark your curiosity and deepen your understanding of the critical topics that will be explored during the conference. These events are your gateway to unparalleled insights and the opportunity to engage with leading minds in the field.

Stay tuned for more information on how you can be a part of these events leading up to the ECRD 2024 on 15-16 May 2024. Prepare to be inspired, informed, and involved – the journey to ECRD is about to get even more exciting!

In the meantime, remember to register for the conference HERE.


Prior to this year’s conference, we’re releasing a number of podcast episodes linked to the themes of some of the parallel sessions taking place. These podcasts will provide an opportunity for listeners to take a deep-dive into the session content and familiarise themselves with key topics before ECRD 2024 begins.

Listen below!


Thought Leader Session: Harnessing Digital Solutions for Rare Diseases

Thursday, 25th April 2024, at 12.00 - 13.15 CET

Programme: Click here to view the session agenda.


This workshop aims to facilitate knowledge exchange, foster collaboration, and inspire action towards leveraging digital solutions for the benefit of individuals living with rare diseases.

Learning Objectives

  • Increased Awareness: Participants will be able to describe the unique challenges faced by individuals with rare diseases and explain the potential of digital solutions to address them.
  • Demonstrated Innovation: Attendees are introduced to innovative digital tools and technologies designed for rare diseases, being able to describe the ways in which diverse approaches can improve patient outcomes and healthcare delivery.
  • Strengthened Advocacy Efforts: Participants are able to explain the value and necessity for policies, funding, and research initiatives that support the advancement of digital healthcare solutions for rare diseases.


  • Jelena Malinina, EURORDIS Data Director
  • Bruno Sarfati, Tekkare
  • Elise de Beauvais, responsible for RDK Application
  • Dr Tudor Groza, European Bioinformatics Institute
  • William Declerck, M.D., E-Health Venture
  • Prof. Lizbeth Goodman, SMARTlab
  • Huw Williams, SMARTlab
  • Irina Kubinschi, MedTech Europe
  • Daniel Lewi, The Cure & Action for Tay-Sachs (CATS) Foundation


Thought Leader Session: Collaborating for Change - Transforming Rare Disease Outcomes through Public-Private Partnerships

Friday, 3rd May 2024, at 15.00 - 16.00 CET

Programme: Click here to view the session agenda.


Rare Diseases pose unique challenges that require collaborative efforts from diverse stakeholders including patients, researchers, clinicians, industry partners, and policymakers. 

This session will delve into the dynamics of such collaborations, particularly focusing on Public-Private Partnerships (PPPs), as an essential avenue for driving innovation, research, and improved outcomes for individuals living with rare diseases.

This session co-powered by Together4RD and the Rare Disease Moonshot aims to present best practice examples, opportunities, needs and difficulties of PPPs in Europe and beyond. 

Learning objectives

  • Participants will be able to explain the importance of PPPs in improving research in Rare Diseases
  • Participants will be exposed to the challenges and opportunities in engaging in PPPs in Rare Diseases
  • Participants will be provided with resources and opportunities to enhance the competitiveness of Europe with more PPPs


  • Matt Boltz-Johnson & Roseline Favresse, EURORDIS
  • Kira Gillet, BeSpoke Gene Therapy Consortium
  • Alexandre Betourné, C-Path Institute
  • Holm Graessner, ERN RND
  • Danielle Dong, Sanofi
  • Sala-Dine Chibout, Novartis
  • Magda Chlebus, EFPIA
  • Sheela Upadhyaya, Chair of Together4RD

Thought Leader Session: The Global Commission to End the Diagnostic Odyssey for Children with a Rare Disease

Tuesday 7th May 2024, at 14.00 - 15.00 CET

Programme: Click here to view the session agenda.


Since 2018, the Global Commission to End the Diagnostic Odyssey for Children with a Rare Disease – a multidisciplinary group of rare disease advocates, researchers, physicians, and technologists – has been working towards its vision of a clear path to a timely, accurate rare disease diagnosis for all children. We are excited to share the Global Commission’s renewed efforts and progress to date.

In this session, Global Commission members and co-chairs will share their newly developed global framework to help guide countries in strengthening their diagnostic efforts. This framework will include a resource compendium with case studies of successful initiatives that have made strides to shorten the time to rare disease diagnosis, highlighting learnings for other countries to draw on. The Global Commission is excited to release these materials at the webinar and will call for others in the rare disease community to submit successful examples that we can include in this framework.

Learning Objectives

  • Summarize the value of the Global Commission and its commitment to end the diagnostic odyssey for children living with a rare disease.
  • Share examples of successful initiatives working to reduce the time to rare disease diagnosis.
  • Explain remaining gaps in the rare disease community and how the Global Commission’s work factors in.


  • Yann Le Cam, EURORDIS, Global Commission Co-Chair
  • Neil Inhaber, Takeda, Global Commission Co-Chair
  • Mike Porath, The Mighty
  • Gareth Baynam, Rare Care Centre, Perth Children’s Hospital
  • Roberto Giugliani, Casa dos Raros, Dasa, HCPA, UFRGS
  • Alexandra Heumber, Rare Disease International
  • Alaa Hamed, Sanofi, Global Commission Co-Chair

Satellite Meeting: The Romanian National Alliance for Rare Diseases - RONARD

Monday, 13th May 2024, at 15.00-17.00 EET (14.00 - 16.00 CET)

Platform: This Satellite Meeting will be taking place on Zoom. Please access it via the following link:

Description & Learning Objectives:

  • Exchange the views of different stakeholders on the links between national and EU policy framework for rare diseases.
  • Raise awareness of the collaborative efforts needed to create clear policy recommendations on a national and EU level.


Session One

  • Dorica Dan, President of RONARD, EURORDIS Vice-President – Moderator
  • Avril Daly, EURORDIS President – Opening Remarks
  • Dr. Diana Păun, Romanian Presidency, State Councilor for Health
  • Alexandru Rafila, Minister of Health, Romania
  • Valeria Herdea, President National Health Insurance House
  • Razvan Prisada, President of the National Agency for Drugs and Medical Technologies
  • Cristian Buşoi, MEP
  • Nicolae Stefănuță, MEP
  • Bianchi Ioana, ARPIM Foreign Affairs Director

Session Two

  • Emilia Severin, Vice-President of the National Council of Rare Diseases, UMVCD – Moderator
  • Maria Puiu, Romanian representative in the Board for ERN of the Member States, UMFT
  • Ioana Streața, project coordinator JARDIN Romania, UMFCV
  • Dorica Dan, President of RONARD, EURORDIS Vice-President
  • Arabela Acălinei, President, EAMDA – European Alliance of Neuromuscular Disorders Associations and Neuro Move CMT Romania
  • Michaela Dan, Romanian Gaucher Association



EJP RD Final Conference

Monday, May 27th (8:30 CET) - Tuesday May 28th 2024 (14:00 CET)

Platform: The conference will be taking on a hybrid format, taking place at both Hotel Excelsior Bari, Via G. Petroni, 15, Bari / Puglia, Italy and online.


This conference is set to bring together leading experts, professionals, and enthusiasts from around the world. As the last EJP RD conference, our objective is to demonstrate the impact of EJP RD activities on the rare diseases community and disseminate all tools that are available to support the whole community.

Find out more about the conference here.

Organised by:

Co-organised by:


With the support of

AFM logo

EU funding logo