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

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.

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.

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.

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.

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.

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.

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.

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.

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

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

'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.
