Webinar - Clinical trials across the lifespan: Considerations for trials with pregnant people and children
2024-09-19 12:00 - 13:00 (America/New_York)
Webinar objectives:
1. Identify reasons why more clinical trials should include children and pregnant people
2. Describe additional considerations for designing, operating, and communicating about clinical trials with pregnant people and children
3. Discuss opportunities and challenges for considering clinical trials across the life span with IMPaCT and PregTrials

'Clinical neurophysiology in dystonia' by Anke Snijders
2024-09-24 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).

Canadian Association of Genetic Counsellors 2024 Annual Education Conference
2024-09-25 08:00 - 2024-09-28 17:00 UTC

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.

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

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)

4th Regional Meeting on ITP - PDSA Canada
2024-09-29 10:00 - 15:00 (America/New_York) | The Courtyard Marriott Burlington, Burlington, Ontario, Canada
On September 29, 2024, the 4th annual Canadian Regional Meeting will take place in Burlington, ON, for patients and families affected by ITP. The event will be held at the Courtyard Marriot. This meeting is a chance to meet other Canadian ITP patients and learn from Canadian ITP experts. If you are interested in registering for the event, be on the lookout for updates on pdsa.org/Canada or reach out to Jennifer DiRaimo at jdiraimo@pdsa.org.

PxP Webinar 3 2024: Different opportunities to engage as a patient partner
2024-10-08 08:00 - 17:00 (America/Chicago)
The webinar series is all about patient engagement in health research, also known as patient and public involvement (UK) or consumer involvement (Australia). It is worth mentioning that ‘patient engagement’ in health research is distinctly different from signing up to be a research participant. Taking part in a study as a participant (for example trying a new treatment or management program) can be a great way to help advance healthcare, but the role would not generally include giving input into the research study itself.

🔵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)

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.

'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)

EURORDIS Mental Health & Wellbeing Open Webinar
2024-10-23 14:00 - 15:30 (Europe/Paris)

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.

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

EDS ECHO Summit: Diet and Nutrition
2024-11-02 09:00 - 18:00 (America/Chicago)
The Ehlers-Danlos Society is delighted to welcome you to register for the EDS ECHO Summit: Diet and Nutrition, a virtual event held on November 2, 2024. This event is suitable for community members, their friends, families, and caregivers, as well as healthcare professionals.

2024 Annual Pain Centre Conference - Building Bridges to Reduce Disparities in Pediatric Pain
2024-11-05 | toronto, ontario, 686 Bay Street, Toronto, Ontario, M5G 0A4, Canada

FSHD Clinical Updates
2024-11-05 17:00 - 18:00 UTC
Muscular Dystrophy Canada (MDC) and the Neuromuscular Disease Network for Canada (NMD4C) are pleased to invite you to a webinar on FSHD clinical updates. This webinar is for Canadian clinicians, academics, researchers, and trainees with an interest in neuromuscular disease. NMD4C and MDC are providing organizational and technical support. This is an independent clinical/academic webinar with no industry sponsorship or influence. AGENDA: Chair and Speaker: Dr. Lawrence Korngut Speakers: Dr. Patrick Horn: Results of a phase 3 study of losmapimod in the treatment of FSHD Dr. Eva Chin: Catalysing Innovation in FSHD Therapeutics Objectives: – Innovative new therapies on the horizon for FSHD – The role of patient foundations and patient-funded R&D for accelerating novel therapies – Understand the outcome to the REACH study of losmapimod in FSHD The NMD Rounds & Educational Webinars is a self-approved group learning activity (Section 1) as defined by the Maintenance of Certification Program of the Royal College of Physicians and Surgeons of Canada. If you have any questions, please email <research@muscle.ca> If you would like to learn more about NMD4C and to become a member: https://neuromuscularnetwork.ca/become-a-member/

Diagnostic approach to childhood-onset chorea' by Juan Darío Ortigoza-Escobar
2024-11-05 19:00 - 20:00 (America/Chicago)
