Canadian Rare Disease Network

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

C-Path 2024 Global Impact Conference
2024-09-09 08:00 - 2024-09-11 17:00 (America/Denver) | Ontario
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.

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.

Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence
2024-09-04 10:00 - 16:00 (America/New_York) | Ontario
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.

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.

WHA Resolution on Rare Diseases Webinar
2024-08-30 14:00 - 15:30 (Europe/Helsinki) | Ontario
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.

Canadian Scleroderma Research Group - Navigating Systemic Sclerosis Waters
2024-08-30 10:30 - 11:30 UTC | Ontario
The CME (Continuing Medical Education) Session hosted at the National Scleroderma Conference is for family practitioners, ER physicians, critical care physicians, internists, rheumatologists, nephrologists, cardiologists, respirologists, nurses, physiotherapists, occupational therapists, respiratory therapists, pharmacists, and students.

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.

Exploring the Brain – Advocating for Your Child with Disabilities
2024-08-28 19:00 - 20:30 UTC | Ontario
Are you a parent, caregiver or educator looking for ways to best support a child or children with disabilities? Join panelists for this upcoming webinar! They’ll cover crucial topics to help you advocate and achieve results for your child. Learn how to navigate the individualized education program (IEP) process effectively; foster a positive, inclusive school experience; and find inclusive community activities and services. They’ll explore community and social opportunities to enhance your child’s development and discuss planning for a successful transition from school to adulthood. Don’t miss this chance to gain valuable insights and connect with experts dedicated to empowering you and your child.

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

CORD Hosts Open Dialogue with Health Canada Webinar
2024-08-21 12:00 - 13:00 (America/Halifax) | Ontario
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

Prioritizing Disability Inclusion in Health Research
2024-08-19 13:00 - 14:00 (America/New_York) | Ontario
Join members of the CHILD-BRIGHT National Youth Advocacy Council on Aug. 19 at 1 p.m. ET/10 a.m. PT as they explore the crucial importance of including people with disabilities in health research focused on equity, diversity, inclusion, decolonization, and Indigenization (EDI-DI). They will define key concepts such as tokenism, infantilization, and non-inclusive environments, leading to a discussion of why inclusion is essential and an overview of elements of inclusive research practice.

Showing Up 101
2024-08-15 15:00 - 16:00 (America/Chicago) | Ontario
“Showing Up 101” is a gently guided, grief informed experience intended to share a language of loss, introduce frameworks for grieving and coping, and promote openness and connection for moving with loss. The content is grounded in bereavement-science, lived experience, and movement-based practices for regulating ourselves. In this 60 minute session, participants will feel equipped and empowered to integrate loss language in their lives, to better communicate how they are feeling to themselves and others, and to understand the power of community for sustaining the hardest, most painful losses. The session is led by Myra Sack, a bereaved mother who is also a Compassionate Bereavement Care Provider, Founder of E-Motion, Inc., and the author of Fifty-Seven Fridays: Losing Our Daughter, Finding Our Way.

Reproductive Health and Autoinflammatory Disease
2024-08-11 11:00 - 12:30 (America/Chicago) | Ontario
Have you ever wondered about how autoinflammatory diseases and their treatments affect your reproductive health?
As part of its Questions, Answered series, the Canadian Autoinflammatory Network – Réseau Auto-inflammatoire Canadien has invited University College London and NHS’ Dr. Helen Lachmann to teach autoinflammatory disease patients about covering conception, pregnancy and breastfeeding in general, as well as specifics concerning some specific drug interactions.

Considerations for effective integration of gender and sex in research and clinical trials
2024-08-07 12:00 - 13:00 (America/Halifax) | Ontario
In this session, Dr. Natalie Rosen will explore best practice guidelines for researchers to build more equitable, reflective, and inclusive research and clinical trials with respect to gender and sex. Dr. Rosen will touch briefly on a variety of topics including, participant eligibility, recruitment, sampling plans and data analysis, measurement of genders and sexes, responsiveness across time, and working with validated scales. The objective is to stimulate critical thought about effective integration of sex and gender in research and clinical trials and to offer some key resources for continued learning.

CHILD-BRIGHT Network - Applying Anti-Racism Principles in Healthcare
2024-07-31 12:00 - 14:00 (America/Halifax) | Ontario
Join us for “Applying Anti-Racism Principles in Health Care,” on July 31, 12:00-2:00 p.m. EST/10:00 a.m.-12:00 p.m. PST. In this interactive workshop, Anna Hossain will be exploring systemic racism in the health care and research sectors.
Key highlights:

Recognize and understand the impacts of unconscious bias in health care interactions and relationships

Understand the foundations of anti-racism and anti-oppression

Gain practical tools to mitigate bias and support inclusive leadership

Platelet Disorder Support Association-ITP Conference 2024
2024-07-26 08:00 - 2024-07-28 17:00 (America/New_York) | The Westin Riverwalk, San Antonio, TX, United States
This year, PDSA will host its 24th annual update on immune thrombocytopenia (ITP) for patients, caregivers and the medical community in San Antonio, Texas July 26-28. This year’s patient conference will take place at The Westin Riverwalk in downtown San Antonio. Each year the ITP conference provides opportunities to hear the latest information about ITP, meet others who are coping and living with ITP and receive answers to your medical questions. You will also have the opportunity to ask your questions to PDSA’s Medical Advisors, who are some of the most experienced ITP doctors in the world. You don’t want to miss out on this tremendous opportunity to hear and talk with these world-renowned experts.
In addition to the educational program and social events, the conference will feature several small group sessions to give you an opportunity to get to know other patients and caregivers in an intimate environment. They will also offer programs for children at our Kids Kamp for children ages 5-12. Like conferences in the past, this year’s conference will provide a separate track for teens and young adults.

Cavernous Malformation Canada - 2024 Patient Conference
2024-07-26 | SickKids Hospital Peter Gilgan Centre for Research and Learning, 170 Elizabeth St Toronto, ON M5G 1E8, Canada, Toronto, Ontario, Canada
This year our patient conference is ONE DAY ONLY, with optional activities happening Saturday.
Keynote by Dr. Brent Derry, many expert presentations, and lunch with the scientists, as well as time to mingle with other patients.

EURO-NMD Gene Therapy Webinar Series - Genetic therapies and therapy developments for rare movement disorders
2024-07-23 15:00 - 16:00 (Europe/Amsterdam) | Ontario
Willeke van Roon-Mom is a full professor of Human Genetics, in particular of translational studies of neurodegenerative disorders. Her work is highly translational in nature, working in close collaboration with clinical departments and industry. Unique patient-driven fund raising initiatives contribute not only financial input, but also patient perspective to research programs in her group. The main topic of her research is autosomal dominant neurodegenerative diseases that have aberrant protein aggregation as a pathological hallmark. She studies molecular disease mechanisms, identifies biomarkers and then uses this knowledge to develop novel therapies with a focus on RNA targeting antisense oligonucleotide therapies. She is the co-founder and co-lead of the Dutch Center for RNA Therapeutics that aims to develop RNA targeting therapies for patients with ultra-rare mutations.

Living with Rare Neuroimmune Conditions: Patient Views & Perspectives
2024-07-20 11:30 - 12:15 (America/Halifax) | Hilton Atlanta, Atlanta, GA, United States
Please join Sumaira Ahmed, NMOSD patient and founder of The Sumaira Foundation, for a panel discussion featuring five patients living with rare neuroimmune conditions. Patient perspectives and lived experiences are essential to guiding R&D efforts and generating real-world evidence for therapeutic advances. This is especially true for rare diseases such as NMOSD, MOGAD, autoimmune encephalitis and stiff person syndrome. Yet far too often, the voices of patients are overlooked, marginalized or excluded. This is an opportunity to hear directly from a diverse group of patients living with these disorders who are also actively advocating for their communities. Sponsored by The Sumaira Foundation.

Racial and Ethnic Diversity in Pediatric Clinical Trials: Current Barriers and a Path Towards Inclusive Clinical Trial Design and Conduct
2024-07-17 12:00 - 13:00 (America/Halifax) | Ontario
Webinar objectives:
1. Understand the role that historical and ongoing marginalization play in research participation
2. Discuss how research findings in homogeneous populations are not generalizable to all children
3. Describe research approaches that can improve diversity and inclusion in pediatric clinical trials

2024 Global Learning Conference
2024-07-17 08:00 - 2024-07-21 17:00 (America/Halifax) | Philadelphia Marriott Downtown, Philadelphia, PA, United States
The Ehlers-Danlos Society is delighted to welcome you to register for our 2024 Global Learning Conference in Philadelphia, Pennsylvania, USA! Ehlers-Danlos Society will be hosting this exciting hybrid event, both in-person and virtually. Join individuals and families with Ehlers-Danlos syndromes (EDS) and hypermobility spectrum disorders (HSD) for up to five days of learning, collaboration, and networking.
The conference theme is “EDS and HSD: From Head to Toe” and will feature presentations, case studies, and panel discussions led by experts on the management of symptoms and comorbidities from head to toe in EDS and HSD. The event is suitable for community members and their friends and families, as well as healthcare professionals.

Sneak Peek into Rare Disease Day 2025
2024-07-17 | Ontario
Join our upcoming webinar for a sneak peek into Rare Disease Day 2025!
On Wednesday 17 July we will be hosting three webinars throughout the day to cater to different time zones, so no matter where you are in the world, you can stay up to date on what to expect for the 2025 campaign.
Learn more about the resources we are revamping and the brand-new downloads we will be producing (with the help of our creative agency and our global partners). We will also be sharing the creative concepts for 2025’s video and poster, so you can start planning how you’d like to use our resources in your national contexts for Rare Disease Day 2025!

RARE Storytelling Webinar
2024-07-16 12:00 - 13:00 (America/New_York) | Ontario
While each rare disease presents unique challenges, the common thread of stories and experiences unites us as a community. Global Genes rare storytelling initiative aims to empower individuals within the community with the tools to share their narratives through various mediums such as blogs, articles, in-person presentations, and podcasts.

Pediatric Inclusion Roundtable - Effective Inclusion of Children Early in Clinical Trials
2024-07-16 08:30 - 17:30 (America/Halifax) | Ontario
Join Leavitt Partners, LLC, and Friedreich’s Ataxia Research Alliance (FARA) for the Pediatric Inclusion Roundtable: Effective Inclusion of Children Early in Clinical Trials. The program will engage all stakeholders regarding the need for including children earlier in clinical development, the requirements for early inclusion, and the most effective means for meeting those requirements.

CADTH: CDA-AMC Webinar – An Update on the Development of Newborn Screening Guidance in Canada
2024-07-11 13:00 - 14:00 (Asia/Dubai) | Ontario
Join Dr. Hilary Vallance and Whitney Ayoub-Goulstone, cochairs of the Newborn Screening Advisory Panel, provide an update on our work to develop pan-Canadian guidance for newborn screening programs across the country in support of the Government of Canada’s National Strategy for Drugs for Rare Diseases. This update will focus on the foundational elements in scope for the advisory panel, including developing a proposed common set of guiding principles for newborn screening, exploring a proposed process and the criteria for the addition or removal of conditions, and recommending conditions that newborn screening programs in Canada could screen.
As part of this event, a discussion paper will be posted on our website with an open call for feedback.

Tell Me More: Why is Obtaining a Diagnosis Challenging?
2024-07-11 12:00 - 13:30 (America/Halifax) | Ontario
We invite you to our next UDNF Tell Me More Lecture Series on Thursday, July 11th at 12:00 p..m. ET for Why is Obtaining a Diagnosis Challenging? – Navigating the Uncharted Waters of an Ultra Rare Disease.
Are you an undiagnosed patient who has been on a long diagnostic odyssey? Come join us for a discussion of the various types of testing used to obtain a diagnosis and their limitations. The complexity of attaining a diagnosis will be explored. Tips to help shorten the diagnostic odyssey will be presented. Lastly, strategies to remain hopeful while living undiagnosed will be shared.

PRISMS Leads the Way To Support Those Living with Smith-Magenis Syndrome at 12th International Conference
2024-07-11 - 2024-07-13 | Hyatt Regency Dallas, Dallas, TX, United States
PRISMS 12th International Conference is the largest conference specifically focused on Smith-
Magenis Syndrome (SMS) in the world. The conference goal is to educate and share information about SMS while providing opportunities for engagement and community amongst SMS families and professionals. This educational and family support multi-day event is a critical program for the SMS community.
The conference theme is “Leading the Way Together” and will share the most recent advances in diagnosis, treatments, and management of SMS throughout the lifespan.
Registration for PRISMS’ 12th International Conference is now open. The conference will be held at the Hyatt Regency in Dallas, TX July 11-14, 2024.

North America Rare Diseases Summit 2024
2024-07-11 | Radisson Blu Aqua Hotel, Chicago, IL, United States
This event brings together leading researchers, healthcare professionals, patient advocates, policymakers, industry experts, and rare disease patients and families. The summit emphasizes the power of collaboration and solidarity in facing rare diseases.

Webinar: Rare Barometer survey on the impact of rare diseases on everyday life
2024-07-10 14:30 - 16:30 (Europe/Helsinki) | Ontario
On Wednesday 10 July, Rare Barometer, the global survey initiative of EURORDIS – Rare Diseases Europe, will launch a new survey on the impact of rare diseases on everyday life.
The survey will be open to all people living with any rare disease and their family members worldwide and will be available in 25 languages, from 10 July to 8 September.
Join us on 10 July for a webinar, when we will discuss the objectives and content of this new survey, how your participation can influence policy and advocacy, and where we will present you with a communication toolkit to help disseminate the survey amongst your networks.
