Webinar - Designing effectiveness-implementation hybrid trials in child health research
2024-09-25 12:00 - 13:00 (America/New_York)
The webinar will introduce effectiveness-hybrid designs, showcase examples, and outline important considerations for conducting these types of studies. It will also briefly summarize tensions that may occur in conducting implementation research and mention tools to support implementation planning and execution.

Turner syndrome and fertility options
2024-09-25 15:00 - 16:00 (America/Chicago)
Register now for a presentation by Professor Janielle van der Velden, paediatric endocrinologist, Radboudumc, Amalia Children’s Hospital, Nijmegen and expert in Turner syndrome.
Event chaired by MTG7 chair Hedi Claahsen, paediatric endocrinologist, Radboudumc, Amalia Children’s Hospital, Nijmegen

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.

Autoimmune Encephalitis and Immune-mediated Epilepsies
2024-09-26 09:00 - 10:30 (America/Chicago)

Western Canadian Neuromuscular Conference (WCNMC)
2024-09-27 17:00 - 2024-09-29 13:00 (America/Chicago) | Hotel Arts Calgary, Calgary, Alberta, Canada
This is the 5th WCNMC and is being held in Calgary on September 27 – 29, 2024. The conference has rotated locations in Western Canada with the inaugural conference hosted in Calgary in 2013. This weekend conference is designed for general neurologists, neuromuscular specialists, physical medicine and rehabilitation specialists, trainee physicians with interest in Neuromuscular Medicine and allied health members from the neuromuscular community.
The conference anticipates 100 – 150 attendees. The majority of attendees will come from Calgary, Edmonton and Vancouver, with a smaller but important complement of colleagues travelling from mid-sized centres such as Victoria, Regina, Kelowna, Lethbridge and Saskatoon.
This conference has been well received in the past within the neuromuscular community as an opportunity to update and share knowledge. The conference has not been held in-person since 2017 and there is much excitement and enthusiasm for this conference within the neuromuscular community to reconnect in person.
The conference will update participants on recent advances in the diagnosis and management of common nerve, muscle and motor neuron diseases. The format will be a blend of short didactic presentations with interactive discussions, international keynote speakers leading the plenary sessions and question answer sessions. Additionally, the conference will provide ample networking opportunities as the exhibit hall will be in the same space as all meals and include a robust welcome reception.
If you have questions about speakers, sponsorship or anything else regarding WCNMC, please contact info@wcnmc.ca.

4th International Symposium on Arthrogryposis
2024-09-29 08:00 - 2024-10-01 17:00 UTC
The International Symposium on Arthrogryposis provides a platform for learning and exchanging on the clinical management and research in Arthrogryposis Multiplex Congenita (AMC) in the areas of genetics, orthopedics, and rehabilitation. Multisite collaborations are key to advance research initiatives and standardize practice across centers.

ERKNet/ESPN Webinar: "Ciliopathies" by Marijn Stokeman
2024-10-08 08:00 - 09:00 UTC

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

Understanding the importance of the social determinants of health for children and youth with disabilities
2024-10-08 12:00 - 13:00 (America/Chicago)

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.

ALS Canada Webinar: Medical Assistance in Dying (MAID) in Canada
2024-10-09 13:00 - 15:00 (America/New_York)

Stem Cell Therapies for Neuromuscular Disorders
2024-10-15 12:00 - 13:00 (America/Chicago)

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.

Supporting the Mental Health Needs of Rare Disease Patients, Families, and Communities
2024-10-16 15:00 - 16:00 (America/Chicago)
During this session, Dr. Al Freedman will provide tools and resources for supporting the mental health needs of rare disease patients, families and communities.

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)

RQMO Educational Day 2024
2024-10-19 08:00 - 17:00 (America/Halifax)
The 2024 RQMO educational day will focus on a theme that affects many families who have a child with a rare disease:
The transition to adulthood of children with differences.

#ChildHealthCan2024
2024-10-20 08:00 - 2024-10-22 21:00 (America/St_Johns) | St.Johns, Newfoundland, 50 New Gower Street, St.Johns, Newfoundland, A1C 1J3, Canada
Children’s Healthcare Canada and co-hosts, the Janeway Children’s Hospital Foundation and NL Health Services, invite you to join us in picturesque Newfoundland and Labrador from October 20-22, at the St. John’s Convention Centre for their 2024 Annual Conference, Right-Sizing Health Systems for Kids: Navigating to Brighter Futures. For those unable to attend in person, a real-time virtual option will also be available.
Children’s Healthcare Canada is on a mission to Right-Size Child Healthcare Systems. In 2024, they will explore how to pave the way for brighter futures for children, youth, and families within the healthcare systems. Through the collective efforts of their members, strategic partners, and sponsors, they are dedicated to ensuring that all children and youth in Canada have access to the healthcare services they require in a timely manner.
We are thrilled to share that we will be hosting another conference in the Spring of 2025, giving attendees the opportunity to engage in two conferences within a six-month period!
Join us at our upcoming conferences in October 2024 and 2025 as they delve into the concept of right-sizing health systems. Together, they will discuss what it takes to establish a flexible and responsive system that meets the unique needs of children and youth, ultimately leading to improved outcomes for all.
Don’t miss the opportunity to contribute to the conversation on shaping a better future for the health and well-being of our youngest generation. They eagerly anticipate welcoming you to St. John’s, Newfoundland and Labrador, as we work towards a brighter tomorrow for our children and families.

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.

Update on treatment in Pompe disease
2024-10-22 15:00 - 16:00 (America/Chicago)
Muscular Dystrophy Canada (MDC) and the Neuromuscular Disease Network for Canada (NMD4C) are pleased to invite you to a webinar on treatment updates in Pompe disease. This case-based rounds webinar is for Canadian clinicians, academics, and trainees with an interest in neuromuscular disease.

'Neurogeriatric and neurological aspects in palliative care of MSA, PSP and advanced PD' by Martin Klietz
2024-10-22 19:00 - 20:00 (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).

The 4th Annual Canadian Children, Youth and Communities (CCYC) Health In | Equity Conference
2024-10-24 08:00 - 2024-10-25 17:00 UTC
The Canadian Children, Youth and Communities (CCYC) In | Equity Conference is an interdisciplinary conference designed to bring together pediatric health and allied health care professionals to address health care inequities from birth to adolescence using a family-centred approach.
This conference explores health equity topics specific to marginalized communities in the Canadian health care system and offers approaches that can be implemented to address barriers in accessing healthcare for under serviced, high-risk and economically disadvantaged children and youth.

Epilepsy Treatments in Rett Syndrome
2024-10-24 13:00 - 14:00 (America/Chicago)
The International Rett Syndrome Foundation (IRSF) invites you to this live RettEd session with a goal of educating families on an approach to the treatment of epilepsy in Rett syndrome

L4L Session: Sickle Cell Disease and Complications including Stroke
2024-10-26 09:00 - 10:00 (America/Chicago)
The SCAGO is excited to announce the October Learning for Life session “Sickle Cell Disease and Complications including Stroke” slated for Sat. October 26th! Attendees will leave the session with a rich knowledge of how to prevent complications of Sickle Cell Disease and improve their health outcomes.

Celebrating 15 Years of TACT: A Milestone Event in Boston
2024-10-31
This year marks the 15th anniversary of the TREAT-NMD Advisory committee for therapeutics (TACT). TACT was set up in 2009 by Prof. Volker Straub and Kate Bushby to provide the neuromuscular community with a unique drug development resource. The aim is to give transparent and consistent guidance to the neuromuscular research community in order to advance the treatment of neuromuscular diseases more effectively. Each year the committee meets in the spring and the autumn, and a bespoke panel is assembled to review up to four applications for advice from organisations developing treatments in NMD.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 ample opportunities to pose questions to the speakers, fostering meaningful dialogue and knowledge exchange.

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.

CRDN Townhall – An Update on the Co-Development of our Strategic Plan
2024-10-31 12:00 - 13:00 (America/Denver)
Join the Canadian Rare Disease Network (CRDN) – Réseau Canadien des Maladies Rares – for a dynamic townhall to engage with thought leaders, experts, and the rare disease community as we explore and shape the future of rare disease care and research in Canada. This townhall will provide an overview of the CRDN and its draft strategic plan. Following the presentation, there will be an opportunity to ask questions and contribute to the co-development of CRDN’s strategic plan. Whether you are a patient, caregiver, researcher, healthcare provider, advocate, or other member of the rare disease community, your voice is vital in building a more integrated, inclusive, and compassionate approach to rare disease care and research in Canada. Register now to be part of this conversation!

Virtual Event: Understanding EDS/HSD+ Symptomology, Diagnoses and Treatment - A Guide for Primary Care Practitioners & Physician Specialists
2024-11-02 08:00 - 17:00 (America/Chicago)
This Conference is for primary care practitioners and physician specialists who encounter patients with complex health issues and chronic pain in their clinical practice, which may or may not be related to Ehlers-Danlos Syndromes or hypermobility Spectrum Disorders (EDS/HSD+).

The Association X Fragile Québec Conference
2024-11-02 08:00 - 18:00 UTC
