13th Conference of Undiagnosed Diseases Network International
2024-09-05 08:00 - 2024-09-07 17:00 (Asia/Tokyo) | Seoul National University Children’s Hospital, Seoul, Korea, Republic of
This event will be held in Seoul, South Korea, and promises to be an enriching experience filled with insightful discussions and valuable networking opportunities.

C-Path 2024 Global Impact Conference
2024-09-09 08:00 - 2024-09-11 17:00 (America/Denver)
Attendees of the 3-day conference, including patients, providers, researchers, clinicians, biopharmaceutical companies, regulatory reviewers and scientists, will leave with knowledge of:

Accelerating drug development across neurology, rare diseases, pediatrics, and beyond.
Integrating insights across therapeutic areas to advance the process for novel treatments and cures.
The power of every precious data point in rare and orphan diseases.
Navigating novel therapies with novel evidence sources.
Innovative methods and approaches.
The value and impact that C-Path partnerships have on accelerating drug development.

31st European Paediatric Rheumatology
2024-09-11 08:00 - 2024-09-14 17:00 (Europe/Paris) | Sweden, Gothenburg, Sweden
As always at PReS meetings, the invited faculty represents internationally renowned clinicians and scientists who will present and discuss significant and exciting developments in pathophysiology, diagnostics, and clinical management of pediatric rheumatology. In the program they will recognize some of the favorites over the year: Grand debates and Thieves market. Added to that many outstanding presentations with varying themes. Also, of course, as tradition is, they will find Young Investigators Meeting and EULAR Pediatric Ultrasound Course.

Global Alliance for Genomics and Health - 12th Plenary
2024-09-16 08:00 - 2024-09-20 17:00 (Australia/Melbourne) | Park Hyatt Melbourne, Melbourne, Australia
GA4GH 12th Plenary brings together organisations and initiatives from the genomics and health community for keynotes, talks, and workshops. The conference will focus on genomic and clinical data sharing issues that pervade diverse industries, disciplines, and communities.

Week in RARE: RARE Advocacy Summit and Health Equity Forum
2024-09-25 08:00 - 2024-09-28 20:30 (Asia/Karachi) | Kansas City, Kansas City, MO, United States
Global Genes is on the move — Week in RARE is moving to Kansas City, Missouri in 2024. Week in RARE combines the RARE Health Equity Forum and RARE Advocacy Summit, in addition to the RARE Champions of Hope awards ceremony and annual membership meetings for Global Advocacy Alliance and RARE Corporate Alliance. This is a unique opportunity to gather and engage rare disease advocates and leaders in the same space for conversation.
Each year, Global Genes convenes one of the world’s largest gatherings of rare disease patients, caregivers, advocates, healthcare professionals, researchers, partners, and allies at the RARE Advocacy Summit to work together to build a path to hope. This is an unparalleled opportunity for advocates, whether new or veteran, to forge meaningful connections with others in the rare disease community for future collaboration. Sessions provide attendees, with insights about the latest in rare disease innovations, best practices for advocating on an individual and organizational level, and actionable strategies they can take home and implement immediately to improve care and accelerate change.
The RARE Health Equity Forum is the first one-of-a-kind event focused on the intersection of rare disease and health equity with an agenda built around creating systemic and organizational change. The conference was developed to better understand the persistent gaps that exist in the rare disease community for underrepresented and/or marginalized patient populations, including those who are still seeking a diagnosis or are unaffiliated with a disease-specific foundation.
This year, the Summit will be held in Kansas City, Missouri.

North American Cystic Fibrosis Conference
2024-09-26 08:00 - 2024-09-28 17:00 (America/Halifax) | Boston Convention and Exhibition Center, Boston, MA, United States
NACFC provides a collaborative and educational forum for all CF professionals. The educational elements of the meeting program are targeted to physicians, nurses, research scientists, respiratory therapists, physical therapists, nutritionists, social workers, and pharmacists.
This annual meeting brings together scientists, clinicians, and caregivers from around the world to discuss and share ideas on the latest advances in CF research, care, and drug development and to exchange ideas about ways to improve the health and quality of life for people with CF.

2024 Global CMT Research Convention
2024-09-26 08:00 - 2024-09-28 17:00 (America/New_York) | Cambridge, Cambridge, MA, United States
The CMT Research Foundation unites the voices of patients, researchers, pharma companies, and regulatory agencies to develop treatments for CMT.
This event takes place over three days. Thursday and Friday are scientific meetings meant for researchers and scientists working in CMT. Saturday is primarily for patients and their families and caregivers, providing a chance to interact with the research community that is working so hard on their behalf and to better understand the latest in research progress.

🔵26èmes Journées Françaises de l’Epilepsie
2024-10-08 08:00 - 2024-10-11 17:00 (Europe/Helsinki)

International Conference on Newborn Screening 2024
2024-10-09 - 2024-10-10
The 2024 meeting of the International Consortium on Newborn Sequencing (ICoNS) will be held in New York City, USA on October 9 – 10, 2024.
ICoNS is the premier event for connecting with top experts and gaining insights into the frontier of newborn sequencing as screening for ushering in the era of preventive medicine.

21st Biennial ESID Meeting
2024-10-16 08:00 - 2024-10-19 17:00 (Europe/Paris) | france, Marseille, France
The ESID 2024 meeting in Marseille will cover the latest most exciting discoveries in basic and clinical science covering our expanding field, bringing together internationally renowned scientists and clinicians to present and discuss major and exciting developments in diagnosis, clinical management, genetics, immunobiology of inborn errors of immunity, through keynote lectures, symposia, educational workshops, interactive sessions, poster presentations, and meet-the-expert sessions.

EPNS Research Meeting 2024
2024-10-17 08:00 - 2024-10-18 17:00 (Europe/Helsinki)
The EPNS has been organising Research Meetings since 2008. With an aim to enhance research in Paediatric Neurology, the meeting brings together leaders in the field, active researchers and young fellows who are interested to join or to set up research projects. It is a forum for researchers in Paediatric Neurology, where they present their work, ideas, results, creating the possibility for good interaction between researchers and premises for networking, future collaborative research in Europe and beyond. Experienced professionals and young researchers get together, in an environment where interactive, vivid discussions can take place around the presented topics.

DIFFERENT FACES OF RARE AND COMPLEX EPILEPSIES
2024-10-17 08:00 - 2024-10-19 17:00 (Europe/Helsinki)

World Orphan Drug Congress 2024
2024-10-22 10:00 - 2024-10-25 18:00 (Europe/Helsinki) | Fira de Barcelona Montjuic, Barcelona, Spain
The World Orphan Drug Congress is the largest, most established European meeting dedicated to orphan drugs. This year the conference will take place in Barcelona, Spain.
From cell & gene therapy, genetic testing, 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.

Developing Therapies in Rare Neuromuscular Disease
2024-10-31
The symposium is designed for industry professionals involved in developing treatments for rare neuromuscular diseases and offers a unique opportunity to learn from past mistakes, engage with leading experts, and gain valuable insights into the challenges and opportunities within the field. Attendees can expect interactive sessions and opportunities to pose questions to the speakers, fostering meaningful dialogue and knowledge exchange.

Global Congress of the GANSID: November 8-9, 2024
2024-11-08 08:00 - 2024-11-09 17:00 UTC
GANSID Congress 2024 is the 2nd Congress of the Global Action Network for Sickle Cell & Other Inherited Blood Disorders (GANSID) taking place virtually to ensure all clinicians, people with hereditary blood disorders, and global advocates can participate from any part of the world.

2024 International Congress for Ataxia Research
2024-11-12 08:00 - 2024-11-15 17:00 UTC
Today marks the opening of the largest-ever scientific conference on ataxia research — the International Congress for Ataxia Research (ICAR) is welcoming a record-setting 600 attendees. The London-based conference runs through to November 15 and will be a comprehensive scientific review of new research into the ataxias, covering topics from advances in genetics and disease mechanisms to therapeutics approaches, as well as a debate on emerging ataxia research topics. ICAR represents a strong collaboration between global advocacy organizations focused on advancing research for ataxia, including co-hosts Ataxia UK, Friedreich’s Ataxia Research Alliance (FARA), National Ataxia Foundation (NAF), and Ataxia Global Initiative (AGI).

Rare Disease Day 2025 webinar: Healthcare professionals
2024-11-13 18:00 - 19:00 (America/Chicago)

'DBS in Dystonia - the network perspective' by Juho Joutsa
2024-11-14 16:00 - 17:00 (Europe/Paris)
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).

4th Annual Symposium for Researchers and Clinicians on Wiskott Aldrich Syndrome
2024-11-18 | San Raffaele Institute, Milan, Italy
The fourth international symposium for researchers and clinicians on Wiskott Aldrich Syndrome is being held in Milan, Italy and offers access to the latest research and analysis related to this rare disease.
Meeting participants will gain valuable insights into innovative perspectives in both basic and clinical research. The scientific programme will draw together experts from around the world to discuss breakthroughs in basic research, advances in clinical practice, novel therapeutic approaches and new insights into stem-cell and cellular therapies. An ideal forum to share knowledge, connect with professional colleagues and grow your professional network.

'Autonomic dysfunction in movement disorders' by Pietro Guaraldi
2024-11-19 15:00 - 16:00 (Europe/Paris)

Webinar on ''mental health and behavioral difficulties in children with rare neonatal surgical disease''
2024-11-20 17:00 - 18:00 (Europe/Paris)
You are invited to join an insightful webinar hosted by ERNICA, eUROGEN, and EURORDIS on Mental health and behavioral difficulties in children with rare neonatal surgical disease: assessment, outcomes and implications for research, policy and practice’.
This interactive session will raise awareness of the mental health and behavioral challenges faced by children with rare neonatal surgical conditions,

Muscle pathology of storage diseases with focus on plyglucosan and lipid storage myopathies
2024-11-21 16:00 - 17:00 (Europe/Paris)
Delivered by Prof. Anders Oldfors (University of Gothenburg, Sweden) on November 21st 2024 at 16:00 Paris Time This is a EURO-NMD webinar in collaboration with ERN-RND (rare neurological disorders) and European Academy of Neurology. READ CAREFULLY please: By registering and attending the webinar you consent to the recording of the webinar and its later diffusion through the EURO-NMD websites using an YouTube Channel.

Genetic Rare Immune Disorders Symposium (GRIDS)
2024-11-24 08:00 - 2024-11-25 17:00 (America/Halifax)
GRIDS brings together world renowned experts from across the globe to discuss recent developments and unique challenges in the field of lysosomal storage disorders and/or rare genetic diseases.

'Differential diagnosis aside from neuroimaging for non-Leukodystrophy white matter diseases' by Nicole Wolf
2024-11-26 15:00 - 16:00 (Europe/Paris)

Kabuki Syndrome Foundation: Annual Research Conference
2024-12-05 08:00 - 17:00 UTC

ERDERA Joint Transnational Call 2025 Information Webinar
2024-12-17 14:00 - 16:00 (Europe/Paris)
This Webinar will:

Provide general information on the call, including its scientific goals, administrative aspects, support services, and patient engagement strategies.
Address your questions through two dedicated Q&A sessions.

ERDERA Joint Transnational Call 2025 Extended Information Webinar
2025-01-14 14:00 - 16:00 (Europe/Paris)
The European Rare Diseases Research Alliance (ERDERA) invites you to the Joint Transnational Call (JTC) 2025 Extended Information Webinar.
Objectives of this webinar:
The webinar scheduled for January 14th aims to provide educational and preparatory support to applicants at the pre-proposal stage, focusing on key elements that enhance the quality of their submissions. The objectives include:
Inform applicants about lessons learned about the evaluation.
Guide applicants with key translational, regulatory, data managing, and methodological considerations for drafting strong pre-proposals.
Address your questions through dedicated Q&A sessions.

2025 International Scientific Conference on Albinism
2025-01-21 08:00 - 2025-01-24 17:00 (Europe/Paris)
The Global Albinism Alliance (GAA) is very pleased to announce the 2025 International Scientific Conference on Albinism – ISCA 2025, that will be held virtually on January 21-24, 2025.
ISCA is THE meeting point worldwide for all scientists interested in sharing and improving knowledge of the biomedical aspects of albinism and, more generally, improving the health of those with albinism. It builds on the momentum created in 2012 with the launch of the EDA (European Days of Albinism) conference series that evolved into ISCA in 2022.

Challenges in the Diagnosis and Treatment of Inflammatory Neuropathies
2025-01-23 16:00 - 17:00 (Europe/Paris)
Delivered by Dr. Filip Eftimov (University Medical Center of Amsterdam, the Netherlands) & Jean-Philippe Plançon (EPODIN – European Patient Organisation for Dysimmune and Inflammatory Neuropathies, France), January 23rd 2025 at 16:00 Paris Time This is a EURO-NMD webinar in collaboration with ERN-RND (rare neurological disorders) and European Academy of Neurology. READ CAREFULLY please: By registering and attending the webinar you consent to the recording of the webinar and its later diffusion through the EURO-NMD websites using an YouTube Channel

The Festival of Genomics & Biodata London
2025-01-29 08:00 - 2025-01-30 17:00 UTC
The UK’s largest life sciences event is back!
Now celebrating its 10th year, the Festival has long been the annual get-together for the UK genomics community. And with more speakers and sessions than ever before, the 2025 edition is bigger and better than ever!
As the genomics sector has expanded, so has its impact. The Festival now covers a diverse array of topics beyond genomics, including biodata, diagnostics, cancer research, healthcare, drug discovery and development, infectious diseases, AI, proteomics, multi-omics, single-cell & spatial analysis and so much more.
