CORD Hosts Open Dialogue with Health Canada Webinar
2024-08-21 12:00 - 13:00 (America/Halifax)
1st Bilateral agreement to deliver on Canada’s promise of accelerating access to therapies for rare disease patients has been signed with BC. Two drugs are listed in the announcement. How will the program work in BC and other provinces? Which provinces are next? Which drugs are next? How will patients and others be involved? Guest Speaker: Daniel MacDonald, Director General, Drugs for Rare Diseases, Strategic Policy Branch, Health Canada

Int'l Autoinflammatory Awareness Month 2024: Patient Care & Advocacy
2024-08-24 09:00 - 12:00 (America/Denver)
Learn about adult and pediatric care for autoinflammatory diseases, research networks, standup comedy, patient advocacy plus breakout rooms.

21st BI-ANNUAL NATIONAL SCLERODERMA CONFERENCE
2024-08-29 08:00 - 2024-08-31 17:00 UTC | St John’s Conference Centre, St John's, Newfoundland, Canada
Held in beautiful St. John’s, NL, the 21st Bi-Annual National Scleroderma Conference offers an information-rich opportunity for people living with scleroderma, their caregivers, family members, friends, and medical professionals.
​Research updates, panel discussions, keynotes, workshops, and other educational sessions are led by leading scleroderma researchers, healthcare professionals and other industry leading professionals. Whether you are newly diagnosed or have had scleroderma for many years, the National Conference is a great way to learn about the disease and become connected with others that are dealing with scleroderma.

WHA Resolution on Rare Diseases Webinar
2024-08-30 14:00 - 15:30 (Europe/Helsinki)
This year, Rare Diseases International has begun advocating for the World Health Assembly to have a Resolution on Rare Diseases in 2025, and we need your help to make it a success. This webinar, co-hosted by RDI the Arab Republic of Egypt, the State of Qatar, and Spain will be an opportunity to explain in more detail the opportunities to improve the lives of persons living with a rare disease (PLWRD) and the need to make the Resolution a reality.

Canada's Drug Agency 2024 Symposium: From Disruption to Opportunity: Embracing Change in Health Care
2024-09-04 08:00 - 2024-09-06 17:00 (America/Halifax) | Shaw Centre, Ottawa, Ontario, Canada
Symposium 2024 will delve into the strategies, technologies, and best practices that can help HTA providers and health care decision-makers navigate disruption and unlock new opportunities, including appropriate use of health technology.
Discover how embracing change can lead to innovative HTA methods, greater equity, enhanced patient care, and more effective use of data and analytics. Network with Canadian and international experts and forge valuable connections that can drive future collaborations and partnerships.

Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence
2024-09-04 10:00 - 16:00 (America/New_York)
The Reagan-Udall Foundation for the FDA, in collaboration with the Bill & Melinda Gates Foundation, will host a hybrid public workshop on “Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence.”
Gene therapies work by replacing, silencing, and/or modifying specific genes within a cell to treat, prevent, or cure a disease, and have been used to treat both inherited diseases such as sickle cell disease and acquired disorders such as leukemia.
This public workshop will provide a baseline about the current state of gene therapy interventions, including approved products and considerations for currently licensed gene therapy products and regulator roles regarding these therapies, particularly in low and middle income countries; explore the “next generation” of gene therapies and the breadth of where those products might be deployed; and discuss the opportunities and needs for regulatory oversight of these products in emerging markets. Speakers will include patient advocates, clinicians, academic researchers, biotech/pharma representatives, and regulators.

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.

ERKNet Webinar: "MGRS" by Roberta Fenoglio
2024-09-10 08:00 - 09:00 (America/Denver)

PxP 2024 Conference (For Patients, By Patients)
2024-09-10 08:00 - 2024-09-12 17:00 UTC
Standing for ‘For Patients, By Patients’, PxP 2024 is entirely led by an international team of patient and public partners and is a Patients Included™ event. Our purpose is to share resources, mentorship and community with patient partners and others involved in health research. Ultimately, our goal is partnering to make research stronger.
This free, virtual event aims to bring you a wealth of knowledge, skills and resources on patient engagement in health research. Patient engagement in health research, also known as patient and public involvement (in the UK) or consumer involvement (in Australia). ‘Patient engagement’ in health research is where patients are research team members. This is different from volunteering to be a research participant where you might help advance research or healthcare, but a research participant generally does not give input into the research study itself. Whatever language you prefer, we warmly welcome everyone to attend.

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.

Designing effectiveness-implementation hybrid trials in child health research
2024-09-11 12:00 - 13:00 (America/Halifax)
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.

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.

EMPOWER & INSPIRE 3RD ANNUAL LEIGH SYNDROME SYMPOSIUM
2024-09-17
Empower & Inspire: Understanding and Accelerating Research for Leigh Syndrome
Tuesday, September 17, 2024
Brought to you by:
​Cure Mito Foundation & integrative Cardiovascular Metabolism and Pathophysiology Laboratory (iCaMP) at Boston University
For details please visit curemito.org/conference

RITA Patient-Centered: Risk of Infection and Immunological Rare Diseases
2024-09-18 10:00 - 11:00 (America/Chicago)
Join the webinar “Risk of Infection and Immunological Rare Diseases,” featuring Dr. Laia Alsina, MD, PhD, Chief of the Clinical Immunology and Primary Immunodeficiencies Reference Unit at Hospital Sant Joan de Déu, Barcelona. This patient-centered webinar, organised by RITA, is part of a new series led by Education WG Chair Jordi Anton, alongside Savino Sciascia and RITA patient groups. Discover insights into the latest research and clinical practices for managing rare immunological conditions.

Rare Disease Day 2025 webinar: Enhancing Accessibility in Physical and Digital Spaces
2024-09-18 14:00 - 15:30 (Europe/Helsinki)
Join on 18 September at 2 pm (CEST, UTC+2) for our Rare Disease Day Webinar, “Enhancing Accessibility in Physical and Digital Spaces.” Discover innovative strategies for making environments more inclusive for people with rare diseases, featuring insights from accessibility experts and patient advocates. Don’t miss this opportunity to engage in meaningful discussions with speakers Adéla Odrihocká (Czechia), James Buller (UK), and Andre Felix (Belgium) on how we can create a more accessible world together.

IPNA Webinar: Chronic kidney disease care in low resource settings: Can we make it a smooth ride for children?
2024-09-19 10:00 - 11:30 (America/Denver)
The webinar will discuss the challenges and barriers to optimal pediatric CKD care in low-resource settings and invite experts to discuss potential solutions to address the inequities in care. The webinar will discuss patient and family perspectives and the need for support.

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

CHAEN / International Guideline Conference
2024-09-20 - 2024-09-22 | Hilton Hotel Toronto, Toronto, Ontario, Canada
The Canadian Hereditary Angioedema Network (CHAEN) is an organization of physicians who treat and/or are interested in Hereditary Angioedema. CHAEN is incorporated under the Canada Not-For-Profit Corporations Act.
CHAEN unites physicians committed to ensuring all HAE patients in Canada have access to excellent care that reflects current management and treatment guidelines, and works to promote research and education.

'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.

Webinar - Designing effectiveness-implementation hybrid trials in child health research
2024-09-25 12:00 - 13:00 (America/New_York)

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
