Celebrate Rare Disease Day: Advancing Clinical Trials Together
2025-02-28 12:00 - 13:30 (Europe/Paris)
On February 28th, in recognition of Rare Disease Day, the European project DREAMS (www.dreamshorizon.eu) invites you to a thought-provoking online webinar: “Clinical Trials in Focus: Innovations and Patient Perspectives in Rare Diseases”. Join leading experts as they explore advancements in clinical trial methodologies, outcome measures, and lessons learned from research in muscular disorders.

RE(ACT) Congress and IRDiRC Conference
2025-03-05 08:00 - 2025-03-07 17:00 (Europe/Brussels) | Brussels, Belgium, Brussels, Belgium
The RE(ACT) Congress and IRDiRC Conference 2025, held jointly by the BLACKSWAN Foundation, the International Rare Diseases Research Consortium (IRDiRC) and the European Rare Diseases Research Alliance (ERDERA). From March 5th to 7th, 2025, we will gather in the vibrant city of Brussels (Belgium) for an unparalleled exchange of knowledge and ideas.
The RE(ACT) Congress will be celebrating its 8th edition, and this convergence serves as a beacon for scientific innovation. Our assembly is comprised of visionary leaders, distinguished experts, and emerging talents from diverse scientific domains, converging to unveil groundbreaking research, foster dialogue, and shape policies crucial to rare disease research.
This edition of RE(ACT) Congress and the 6th IRDiRC in person Conference, is in partnership with the European Rare Diseases Research Alliance (ERDERA, planned to start in September 2024), EURORDIS-Rare Diseases Europe, and Rare Diseases International (RDI), and symbolizes a unified commitment to surmounting rare disease challenges through collective action and shared knowledge.

Navigating the Transition from Pediatric to Adult Neuroimmunology Care
2025-03-06 14:00 - 15:00 (America/New_York)
This webinar will provide an overview of the transition from pediatric to adult medical care, featuring insights from a pediatric neurologist, social worker, and a neuropsychologist.
Topics will include maintaining continuity of care, managing complex medical conditions, self-advocacy in healthcare settings, and addressing social and emotional considerations. The discussion aims to support patients, families, and caregivers in navigating this important transition effectively.

Empowering Patients 2025: A Cell and Gene Therapies Summit
2025-03-12 08:00 - 2025-03-13 17:00 (America/Denver)
This free, virtual event in two half-day sessions offers educational discussions for foundation leaders, patients, and caregivers to better understand #GeneTherapy and #CellTherapy.

MDA Clinical and Scientific Conference
2025-03-16 08:00 - 2025-03-19 17:00 (America/Chicago)

NMOSD in sub-Saharan Africa
2025-03-17 18:00 - 19:30 (Europe/Paris)
The experts will provide insights and answer questions about the prevalence and incidence of NMOSD, diagnostics, available treatments, immunizations, clinical trials, and more. Patients, caregivers, clinicians, researchers, and advocates from around the world are invited to attend.
Live participants will have the opportunity to ask questions directly to the experts. The webinar will be recorded and made available for replay on TSF’s YouTube channel.

Webinar on “Neurogeriatric Aspects of Huntington’s Disease and Chorea”
2025-03-18 15:00 - 16:00 (America/Chicago)
Educational webinars on rare neurological, neuromuscular and movement disorders jointly organized by the European Reference Networks for Rare Neurological Diseases (ERN-RND) and Neuromuscular Diseases (ERN-EuroNMD) and the European Academy of Neurology (EAN).

Peripheral Nerve Imaging with Focus on Inflammatory Neuropathies
2025-03-20 16:00 - 17:00 (Europe/Paris)

13th ISNS European Regional Meeting Luxembourg
2025-03-23 08:00 - 2025-03-26 17:00 (Europe/Paris)
Newborn Screening LIVE from the Heart of Europe

Dragon Claw Friends Webinar - Breaking Down Barriers – Are Patients Aware of Their Autoimmune Disease Treatment Options?
2025-03-27 10:00 - 11:00 UTC
Despite significant advancements in the treatment of autoimmune diseases, many patients remain unaware of their options. Social media platforms are filled with stories of people struggling with uncontrolled symptoms—so why aren’t these breakthroughs reaching those who need them most? To explore this issue, Dragon Claw Charity Australia, in collaboration with CaPPRe, has conducted research into the awareness gap among patients with rheumatology, dermatology, and gastroenterology-related autoimmune diseases.

Navigating Life with Dysimmune and Inflammatory Neuropathies: Q&A Session on Quality of Life and Management
2025-03-27 16:00 - 17:00 (Europe/Paris)

13th ISSAID Meeting
2025-04-08 08:00 - 2025-04-10 17:00 (Europe/Helsinki) | paris, Paris, France
13th Meeting of the International Society of Systemic Autoinflammatory Diseases (ISSAID), which will take place in Paris from April 8th to 10th, 2025, at the “Cité Internationale” – a magnificent haven of peace near many transport amenities where you will particularly enjoy the sumptuous Honorat lounge overlooking a lush green park.
April in Paris offers a delightful mix of mild weather, budding greenery, and a relaxed atmosphere, perfect for strolling along the Seine after a day of work.
The ISSAID 2025 meeting will provide an opportunity for pediatric and adult rheumatologists, immunologists, general pediatricians and internists, scientists, and others interested in systemic autoinflammatory diseases to come together to share new findings related to autoinflammation and to learn about recent advances in this field.
The ISSAID 2025 meeting will highlight the topic of neurologic inflammation, and we hope it will facilitate better assessment, care, and research on this particularly complex subject. We will present current topics such as artificial intelligence, mechanisms of inflammation, and new diseases.
The ISSAID 2025 meeting will promote interactions between old and new generations so that the science of SAIDs can be transmitted and best adapted to societal transformations and the challenges of tomorrow’s medicine.

RDI: Leveraging the EML for Access to Rare Disease Treatments
2025-04-10 14:00 - 15:00 UTC
The WHO Essential Medicines List (EML) is a critical instrument for advancing Universal Health Coverage (UHC) by ensuring equitable access to essential medicines. However, its role in rare disease treatment remains under-recognized. This webinar will explore how the EML can serve as a powerful tool to improve access to treatments for persons living with a rare disease (PLWRD) at both global and national levels.
Webinar Objectives:

Gain a deeper understanding of the WHO Essential Medicines List and its significance for rare diseases
Learn about the findings from RDI’s Essential Medicine Working Group on challenges, best practices, and opportunities for regarding the application process of EML
Hear about call-to-action to enhance global and national access to essential medicines

Neuropsychiatry & Mental Health 2025
2025-04-24 08:00 - 2025-04-25 17:00 UTC
Theme: Innovations in Neuropsychiatry and Evolution in Medicines of Neurological Disorders for Better Mental Health.

Mitochondrial Transplantation and Next Generation Therapeutics Conference
2025-04-27 08:00 - 2025-04-29 17:00 (America/Halifax)
Join Northwell Health on April 27-29, 2025, at Hofstra University on Long Island (NY) for a unique forum for the presentation and discussion of new research developments in mitochondrial transfer and transplantation.

Summit 2025: Ending The Diagnostic Odyssey
2025-04-28 08:00 - 2025-04-30 17:00 UTC | washington, WA, United States
A first-of-its kind collaborative event for Undiagnosed, Ultra-Rare & Rare disease industry, clinical and community leaders.
The diagnostic odyssey for rare diseases is a significant challenge, often leaving patients and families lost in a maze of tests, misdiagnoses, missed opportunities to participate in clinical trials, and delayed treatment. Summit 2025 brings together a diverse group of stakeholders – patient advocates, industry leaders, policymakers, clinicians, researchers and payers – to chart a collaborative path towards a future where timely and accurate diagnoses are a reality for all with rare diseases.

Neurodiversity in the Workplace: 6 Principles for Inclusion
2025-04-29 10:00 - 11:00 (America/Edmonton)
Learn how to create a more inclusive workplace for neurodivergent employees with our 6 principles!

Myotonic Dystrophy Foundation Conference 2025
2025-05-01 08:00 - 2025-05-04 17:00 (America/New_York)
MDF invites you to attend the Professional Track at the 2025 MDF Conference in Indianapolis, Indiana from May 1st to May 4th! Sometimes called the “DM family reunion” – the MDF Conference is an event to unite the DM community in our journey towards treatments and a cure for myotonic dystrophy (DM). This year’s program includes educational sessions exploring research, drug development, symptom management, and other critical topics for DM families, plus opportunities for formal and informal networking, wellness, and fun!

15th MGFA International Conference on Myasthenia and Related Disorders
2025-05-13 08:00 - 2025-05-15 17:00 (Europe/Paris)
Hosted by the MGFA, this influential gathering of neuromuscular researchers and medical professionals is a prominent opportunity to present your work and hear from accomplished peers from around the world. We hope that you will be able to join us for this premier scientific gathering of global MG leaders.

Milestones & Momentum: 10 Years of Progress, 10 Years of Possibility
2025-05-21 18:00 - 20:30 (Europe/Paris)
RDI will be holding a side event at World Health Assembly 78 in Geneva. Join them as the community celebrates 10 years of progress and looks forward to 10 more years of redefining what’s possible!

CureDuchenne 2025 FUTURES National Conference
2025-05-22 08:00 - 2025-05-25 17:00 (America/Kentucky/Louisville)
This annual event is dedicated to providing education, connection, and hope to the entire Duchenne community.

European Society of Human Genetics Conference
2025-05-24 08:00 - 2025-05-27 17:00 (Europe/Vienna)

Advancing Innovation in Rare Disease Research
2025-06-02 08:00 - 2025-06-03 17:00 (America/Vancouver)
Join researchers, clinicians, and industry leaders to discuss real-world research breakthroughs, novel #ClinicalTrial designs, and how NORD’s Rare Disease Centers of Excellence are leading collaborative efforts in #RareDisease innovation.

Superficial Siderosis Symposium 2025
2025-06-11 08:00 - 2025-06-13 17:00 (America/Halifax)
Join the Superficial Siderosis Research Alliance (SSRA) for a three-day symposium dedicated to patients, caregivers, clinicians, and researchers focused on advancing care, treatment, and research for superficial siderosis.

Community Conversation: The Living Rare Study
2025-06-12 16:00 - 17:00 (America/Edmonton)
Join NORD on Thursday, June 12 at 4 p.m. ET for a virtual conversation about the #LivingRareStudy. This session will guide you through joining the first large-scale study in the U.S. focused on tracking the real-life experiences of individuals and caregivers impacted by #RareDiseases, including how to create an #IAMRARE account, review the consent form, and navigate the platform.

FSHD Connect Europe patient meeting 2025​​​​​​​​​​​​​​​​​
2025-06-13 08:00 - 2025-06-15 17:00 (Atlantic/Azores)
FSHD Europe will organize, in collaboration with the FSHD Society, the first FSHD Connect Europe meeting on June 13th – June 15th, 2025, in Amsterdam, The Netherlands. This is a networking meeting for FSHD patients and their families. It is a great opportunity to connect with other patients and families across Europe.

The Cambridge Rare Disease Showcase
2025-06-18 18:30 - 21:00 (Europe/London)
This annual event celebrates the innovative work and powerful stories emerging from the rare disease community – uniting patient groups, researchers, clinicians, industry professionals, and more from across the UK and beyond.

Mito Med 2025 Masterclass on Primary Mitochondrial Myopathy with a Focus on TK2d
2025-06-21 08:00 - 17:00 (America/Kentucky/Louisville)
New this year for Clinical-Scientific Attendees to UMDF’s #MitoMed Conference — a Masterclass on Primary Mitochondrial Myopathy (#PMM) with a Focus on #TK2d. Tracks for Early Career Investigators and for Up-and-Coming Clinicians.

THE FESTIVAL OF GENOMICS & BIODATA Boston
2025-06-24 08:00 - 2025-06-25 17:00 (America/New_York)
The Festival is designed principally for scientists and clinicians who are working in the fields of genomics, other omics, and/or using biodata , to further research, drug discovery, healthcare and – ultimately – deliver better patient outcomes.

Topics covered include genomics, single cell/spatial biology, multiomics, biodata, cancer research, drug discovery, AI, microbiome, proteomics, liquid biopsy and so much more.

United in Hope: 2025 Global Prader-Willi Syndrome Conference
2025-06-24 08:00 - 2025-06-28 17:00 (America/Denver)
