30th Annual International Congress of the World Muscle Society
2025-10-07 08:00 - 2025-10-11 17:00 (Europe/Vienna)
The World Muscle Society is a dynamic community that aims to promote, disseminate, and share all aspects of neuromuscular physiology and diseases, from basic science to patient care. It encompasses a broad range of scientists and healthcare professionals who share the common interest for neuromuscular disorders.

Rare Diseases International-Community Conversations on Mental Health
2025-10-10 07:00 - 08:00 (America/Edmonton)

International Neurology Conference 2025 | Mental Health Conference, Thailand
2025-10-18 08:00 - 2025-10-19 17:00 (Asia/Krasnoyarsk)
Neurology & Mental Health conference 2025 is set to be held on the 18th and 19th of October 2025, in Bangkok, Thailand in hybrid mode, allowing everyone to gain knowledge and providing a platform for discussing the latest advancements in treatments and techniques, future and present challenges, and sharing invaluable experiences in Neurology and allied disciplines. The event will feature plenary lectures, speeches, research & technical presentations, and hands-on experience workshops to help shape the future of neurology & psychiatry.

The World Orphan Drug Congress
2025-10-27 08:00 - 2025-10-29 17:00 (Europe/Amsterdam)
The World Orphan Drug Congress is the largest and most established orphan drug & rare disease meeting of its kind across the globe.
From cell and gene therapy, genetic testing, and market access, to real world evidence, this one meeting covers the whole orphan drugs value chain where science, government and manufacturers all come together to create groundbreaking progress.

Screen4Rare: Making AI Diagnosis Understandable
2025-11-04 05:30 - 07:30 (America/Edmonton)
A co-creation workshop inviting non-AI experts to help design communication tools such as videos and brochures that make AI-supported diagnosis more understandable, trustworthy and human.

Webinar: Health Financing & Social Health Protection for Rare Diseases
2025-11-05 06:00 - 08:00 (America/Edmonton)
Rare Diseases International (RDI) and the P4H Network have partnered to enable global advocacy and collaboration to influence and support innovation in health financing arrangement and social health protection that concretely promote effective health coverage, increased access and adequate financial protection for people living with rare diseases.

IPIC 2025
2025-11-05 08:00 - 2025-11-07 17:00 (Europe/Helsinki)
IPIC is one of the leading international congresses on primary immunodeficiency (PID) diagnosis and clinical care with a unique approach to its scientific programme underpinned by its focus on patient-centredness. With a truly international impact, the congress has earned its definite place and role in the PID clinical landscape.

42nd International Conference on Advances in Psychiatry and Mental Health
2025-11-10 08:00 - 2025-11-11 17:00 (Asia/Dubai)
This premier event will bring together leading experts, researchers, and mental health professionals to explore innovative approaches and emerging trends in the field of psychiatry. Psychiatry Conference 2025 cordially invites all psychiatrists, psychologists, researchers, clinicians, mental health professionals, neuroscientists, healthcare practitioners, academicians, and industry experts to join us on November 10-11, 2025, in Dubai, UAE.

3rd Edition on International Neurology Conference 2025 in USA
2025-11-15 08:00 - 2025-11-16 17:00 (America/New_York)

ITINERARE Symposium 2025
2025-11-18 08:00 - 17:00 (Europe/Zurich)
The programme features experts from a variety of fields, ranging from basic and translational research to clinical application and the ethical, social and legal aspects of rare disease research.

36th International Symposium on ALS/MND
2025-12-05 08:00 - 2025-12-07 17:00 (America/Los_Angeles)
The International Symposium is the biggest annual conference dedicated to ALS and MND research. Each year they bring together researchers from around the world to share new understanding of amyotrophic lateral sclerosis and motor neurone disease.

Webinar: 'The importance of genetic counselling in the diagnostic of rare neurological disorders' by Maria Judit Molnar
2025-12-09 07:00 - 09:00 (America/Edmonton)
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).

2nd International Conference on Clinical Research Networks (CRNs) for Rare Diseases
2025-12-09 08:00 - 2025-12-10 17:00 (America/Edmonton)
This second edition will convene a wide range of international experts, including researchers, clinicians, patient advocates, industry representatives, policymakers, and funders to strengthen the collaborative ecosystem that underpins clinical research in rare diseases worldwide.

ERDERA: Joint Transnational Call 2026
2025-12-16 15:00 - 17:00 (America/Edmonton)
This information webinar on the 2026 Joint Transnational Call for Proposals is scheduled for 16 December 2025, 15:00–17:00 CET, focusing on resolving unsolved rare genetic and non‑genetic diseases.

Webinar: Lymphodepletion for CAR-T/IEC Outcomes
2025-12-17 12:00 - 14:00 (America/Toronto)
Achieving consistent and effective outcomes in CAR T and other immune effector cell (IEC) therapies relies heavily on getting lymphodepletion “right.” Emerging evidence shows that both under- and over-exposure can impact safety, efficacy, and long-term persistence, making optimized dosing strategies increasingly critical as the field evolves.
This webinar will explore how model-informed and data-driven approaches can help identify the optimal lymphodepletion exposure window, improve predictability, and support more standardized practices across patient populations and treatment centers. Speakers will discuss current challenges, scientific rationale, clinical insights, and practical considerations for implementation.

CMT episode 1: Challenges in the diagnosis and treatment of inherited neuropathies
2026-01-08 08:00 - 09:00 (America/Edmonton)
Delivered by Dr. Davide Pareyson (Fondazione IRCCS Istituto Neurologico C.Besta (FINCB), Milan, Italy) & Dr. Filippo Genovese (The European Charcot-Marie-Tooth Federation & ACMT-Rete per la malattia di Charcot-Marie-Tooth OdV), Thu 08 Jan 2026 at 16:00 Paris Time This is a EURO-NMD webinar in collaboration with ERN-RND (rare neurological disorders) and European Academy of Neurology.

RealiseD Webinar 1: Realising Clinical trials in Ultra-Rare diseases
2026-01-13 17:00 - 18:30 (America/Edmonton)
Join RealiseD’s first webinar to learn how RealiseD is pioneering a collaborative and comprehensive approach to clinical trials in ultra-rare diseases. During the webinar, they’ll walk you through the project’s objectives, structure, and methodologies—and show how you can be part of this transformative journey.

Rare Disease Internation-Moving the WHA Resolution on Rare Diseases Forward: From Promise to Action
2026-01-15 05:30 - 06:30 (America/Edmonton)
Join RDI for this public webinar where they will discuss the current status of the WHA Resolution implementation, as they approach the World Health Organization Executive Board meeting taking place in February 2026. This webinar will engage in a multistakeholder dialogue focusing on the necessary path forward to accelerate the implementation of the Resolution and importantly the development of the GAPRD.

CMT episode 2: Progress in genetic diagnosis for neuropathies
2026-01-15 08:00 - 09:00 (America/Edmonton)
Delivered by Dr. Andrea Cortese (University College London, Department of Neuromuscular Diseases, UK), Thu 15 Jan 2026 at 16:00 Paris Time This is a EURO-NMD webinar in collaboration with ERN-RND (rare neurological disorders) and European Academy of Neurology.

RealiseD Webinar 2: Evidence Assessment Framework. A need to change mindset from developers, regulators and HTA
2026-01-20 17:00 - 18:30 (America/Edmonton)
The second webinar turns the spotlight on one of the most critical issues in rare disease research: the generation and assessment of evidence. François Meyer, a leading consultant in health technology assessment, will examine why current frameworks often fail to deliver predictable and aligned outcomes, resulting in delays and inequalities in patient access. The session will explore the global and European initiatives aimed at addressing these shortcomings and present the RealiseD consortium’s efforts to advance scientific and operational solutions. More importantly, it will advocate for a fundamental shift in mindset—one that embraces flexibility and innovation in the way evidence is generated, interpreted, and assessed.

CMT episode 3: Bioarkers and outcome measures for clinical trial readiness in CMT
2026-01-22 08:00 - 09:00 (America/Edmonton)
Delivered by Prof. Mary Reilly (University College London, Department of Neuromuscular Diseases, UK), Thu 22 Jan 2026 at 17:00 Paris Time This is a EURO-NMD webinar in collaboration with ERN-RND (rare neurological disorders) and European Academy of Neurology.

ERN-EuroBloodNet-Session 1: Strong at Any Age – Redefining Frailty Together
2026-01-22 17:30 - 18:30 (Europe/Belgrade)
EuroBloodNet Topic on Focus: Ageing with a Bleeding Disorder”!
This educational program is coordinated by ERN-EuroBloodNet together with the umbrella patient organisation EHC – European Haemophilia Consortium, represented by ePAG advocate Marius Tanase.

CMT episode 4: Current Progress in Charcot-Marie-Tooth disease (CMT) Treatments
2026-01-29 08:00 - 09:00 (America/Edmonton)
Delivered by Prof. Kleopas Kleopa (Cyprus Institute of Neurology and Genetics (CING), Thu 29 Jan 2026 at 16:00 Paris Time This is a EURO-NMD webinar in collaboration with ERN-RND (rare neurological disorders) and European Academy of Neurology.

RealiseD Webinar 3: Single arm, RCT or something in between – how to enrich clinical trial design and analysis in rare diseases
2026-02-03 17:00 - 18:30 (America/Edmonton)
Single-arm trials remain a cornerstone of rare disease research, but their limitations and complexities cannot be ignored. In the third webinar, experts Yulia Dyachkova (Merck Healthcare), Jenny Devenport (Roche), Cornelia Dunger-Baldauf (Novartis) abd Guillaume Canaud will lead a deep dive into the evolving role of SATs in drug development. The discussion will cover regulatory and HTA perspectives, review case studies of success and failure, and explore advanced statistical methods designed to optimise trial design. This session is not just about understanding the current landscape—it is about shaping the future of evidence generation for rare and ultra-rare diseases.

8th TREAT-NMD® International Conference – Lisbon, 2026
2026-02-10 08:00 - 2026-02-12 17:00 (Europe/Lisbon)
Join TREAT-NMD at this vital, in-person gathering where patients, clinicians, researchers, industry, and regulators come together to tackle the biggest challenges in neuromuscular treatment development, diagnosis and care. Topics will include; disrupted clinical trial delivery, regulatory uncertainty, global access inequalities and shrinking public funding, they’ll tackle these themes with a holistic, solutions-driven approach and a patient centric and global focus.

RealiseD Webinar 4: Enhancing patient-centricity in Rare Disease Clinical Trials
2026-02-10 17:00 - 18:30 (America/Edmonton)
The final webinar focuses on what truly matters: the patient. Rare disease trials often struggle to capture outcomes that reflect patient priorities but RealiseD is working to change that. Rudradev Sengupta, Senior Trial Design Lead at One2Treat, will present an approach that embeds patient voices into every stage of trial design and analysis. By using hierarchical endpoints and net treatment benefit, this framework balances benefits and risks in alignment with patient preferences, enabling transparent discussions among stakeholders and ensuring that success is defined by what matters most to patients. This is a vital conversation for anyone committed to making clinical research more humane and impactful.

Rare Disease Day 2026: Advancing the Rare Brain Disease Ecosystem
2026-02-24 09:00 - 16:00 (Europe/Brussels)
Placing brain health at the core of rare brain disease prevention and management, this event gathers multidisciplinary experts from research, clinical care, patient advocacy, and industry to share perspectives and priorities on the rare brain disease research, care, and policy space. The meeting will focus on reviewing key developments in regulatory frameworks and discussing strategies to advance the RBD Ecosystem and develop a comprehensive Knowledge Hub.

EP PerMed Hackathon
2026-03-03 08:00 - 2026-03-04 17:00 (America/Guadeloupe)
This is your chance to be a part of an immersive event at the forefront of innovation in personalised medicine. Join ER PerMed and work together with experts, innovators, and visionaries to shape the future of healthcare.

Rare Kidney Disease Foundation: 5th International ADTKD Summit
2026-03-06 10:00 - 2026-03-07 13:00 (America/Toronto)
The Rare Kidney Disease Foundation presents: RKDF 5th International ADTKD Summit – A Time For Action Friday, March 6, 10AM – 1:00PM EST – Science Program Saturday, March 7, 10AM – 1:00PM EST – Patient Program

Rare Disease Summit: Access USA
2026-03-17 08:00 - 2026-03-19 17:00 (America/Edmonton)
The Rare Disease Summit unites stakeholders to accelerate therapeutic innovation for areas of unmet medical need. Gain actionable insights on launch planning, market access, reimbursement strategies and strategic partnerships to optimize rare disease commercialization and elevate patient impact.
