Canadian Rare Disease Network

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

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.

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.

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.

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

CANPKU+ 2024 ANNUAL GENERAL MEETING
2024-09-16 19:00 - 20:00 (America/New_York)
Please note only those who are current members will have the availability to vote at the upcoming AGM. Please check your profile to see if your membership is current.
Not current? Sign up to become a member or renew your membership at www.canpku.org/membership

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.

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.

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.

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.

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

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.

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)
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)
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
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
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)
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)
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)
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)
“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)
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)
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)
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)
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.
