Child Health Hub in Transition to Adult Healthcare
2024-12-02 08:00 - 2024-12-03 17:00 (America/Denver)
The Child Health Hub in Transition to Adult Healthcare is excited to be hosting the 2nd Transition to Adulthood Pop-Up event on December 2-3, 2023, at the beautiful Hyatt Regency in downtown Vancouver. This event aims to bring together youth, families, trainees, researchers, clinicians, and policy/decision makers to celebrate the best of what we are learning, and doing, to help young people to develop fully, even when they are challenged by health conditions. For those unable to join in-person, aspects of the event will be made available in a virtual format.

Duchenne Patient Academy 2024!
2024-12-06 08:00 - 2024-12-07 17:00 UTC
This year, the 8th edition of Duchenne Patient Academy will take place. The Academy provides online training for Duchenne and Becker muscular dystrophy (DMD/BMD) patient advocates.
Duchenne Patient Academy 2024 will take place on Friday December 6, and Saturday December 7 fully online.
Over the course of 2 days, Duchenne Patient Academy attendees will listen to speakers and attend panel discussions moderated by experts in their respective fields. As with last year, attendees have the opportunity to become involved in the dialogue and share their experiences.

Canadian Fragile X Research Rounds: Protein Replacement Therapy for Fragile X Syndrome
2024-12-12 12:00 - 13:00 (America/Denver)

CPD-Accredited Webinar | Exploring the clinical trial landscape in Duchenne muscular dystrophy: Challenges, opportunities and consideration of a possible cholesterol metabolism-related target
2025-01-14 18:00 - 19:00 (America/New_York)
NMD4C and MDC are pleased to invite you to a webinar on Exploring the clinical trial landscape in Duchenne muscular dystrophy: Challenges, opportunities and consideration of a possible cholesterol metabolism-related target. NMD4C and MDC are providing organizational and technical support. This is a an independent clinical/academic webinar with no industry sponsorship or influence.

The Festival of Genomics & Biodata London
2025-01-29 08:00 - 2025-01-30 17:00 UTC
The UK’s largest life sciences event is back!
Now celebrating its 10th year, the Festival has long been the annual get-together for the UK genomics community. And with more speakers and sessions than ever before, the 2025 edition is bigger and better than ever!
As the genomics sector has expanded, so has its impact. The Festival now covers a diverse array of topics beyond genomics, including biodata, diagnostics, cancer research, healthcare, drug discovery and development, infectious diseases, AI, proteomics, multi-omics, single-cell & spatial analysis and so much more.

PKU CON 2025
2025-02-14 08:00 - 2025-02-15 17:00 (Europe/Helsinki) | milan, italy, Milan, Italy
PKU CON aims to bring together all stakeholders from around the globe currently involved in the management of patients affected by this inborn error of metabolism with an unique and same, single goal: optimize the care and assistance for patients affected by Phenylketonuria.
Many topics will be discussed during this first edition, starting from a renewed vision of the pathology, with possible identification of new biomarkers and metabolic control indices, to updates in the field of dietary and pharmacological interventions, and addressing relevant assistance-related issues such as burden of illness and unmet needs.

2025 Clinical Trials Training Summit
2025-02-24 08:00 - 2025-02-26 17:00 (America/Chicago)

From Grassroots to Global: Growing Momentum Towards a WHA Resolution on Rare Diseases
2025-02-27 08:00 - 17:00 (Europe/Paris)
Join RDI for a webinar on the global movement behind the efforts to advocate for a WHA Resolution on Rare Diseases

Rare Disease Day 2025 University of Alberta
2025-02-28 08:00 - 16:00 (America/Denver)

CORD Rare Disease Day 2025 Webinar
2025-02-28 12:00 - 13:00 (America/New_York)
CORD is hosting a special webinar on Friday, Feb 28 from 12-1pm EST so that everyone from across Canada can tune in and help put that funding to work. Here’s what they’ll discuss: – Ending the diagnostic odyssey with CHEO’s ThinkRare – Treating and tracking rare diseases at BC Cancer – Caring for complex kids and adults at BC Children’s Hospital – The challenge of treating adults with SMA in Manitoba – Measuring the socioeconomic impact of rare diseases on families and health systems

RE(ACT) Congress and IRDiRC Conference
2025-03-05 08:00 - 2025-03-07 17:00 (Europe/Brussels) | Brussels, Belgium, Brussels, Belgium
The RE(ACT) Congress and IRDiRC Conference 2025, held jointly by the BLACKSWAN Foundation, the International Rare Diseases Research Consortium (IRDiRC) and the European Rare Diseases Research Alliance (ERDERA). From March 5th to 7th, 2025, we will gather in the vibrant city of Brussels (Belgium) for an unparalleled exchange of knowledge and ideas.
The RE(ACT) Congress will be celebrating its 8th edition, and this convergence serves as a beacon for scientific innovation. Our assembly is comprised of visionary leaders, distinguished experts, and emerging talents from diverse scientific domains, converging to unveil groundbreaking research, foster dialogue, and shape policies crucial to rare disease research.
This edition of RE(ACT) Congress and the 6th IRDiRC in person Conference, is in partnership with the European Rare Diseases Research Alliance (ERDERA, planned to start in September 2024), EURORDIS-Rare Diseases Europe, and Rare Diseases International (RDI), and symbolizes a unified commitment to surmounting rare disease challenges through collective action and shared knowledge.

13th ISNS European Regional Meeting Luxembourg
2025-03-23 08:00 - 2025-03-26 17:00 (Europe/Paris)
Newborn Screening LIVE from the Heart of Europe

Save-The-Date: RareKids-CAN Virtual Information Session
2025-03-24 08:00 - 11:00 (America/New_York)
During this information session, you will gain an overview of RareKids-CAN, build connections within the community, and more!

RareKids-CAN Annual Conference
2025-04-10 08:00 - 2025-04-11 17:00 (America/New_York)
The mission of the RareKids-CAN: Pediatric Rare Disease Clinical Trials and Treatment Network Strategic Planning Conference is to collaboratively shape the future of pediatric rare disease research and clinical trial capacity in Canada. By bringing together over 100 patient and family partners, researchers, sub-platform leaders, and key stakeholders, they aim to co-develop a shared vision that accelerates pediatric drug development and progress in clinical trials, improves treatment access, and strengthens national and international partnerships.

Mitochondrial Transplantation and Next Generation Therapeutics Conference
2025-04-27 08:00 - 2025-04-29 17:00 (America/Halifax)
Join Northwell Health on April 27-29, 2025, at Hofstra University on Long Island (NY) for a unique forum for the presentation and discussion of new research developments in mitochondrial transfer and transplantation.

Summit 2025: Ending The Diagnostic Odyssey
2025-04-28 08:00 - 2025-04-30 17:00 UTC | washington, WA, United States
A first-of-its kind collaborative event for Undiagnosed, Ultra-Rare & Rare disease industry, clinical and community leaders.
The diagnostic odyssey for rare diseases is a significant challenge, often leaving patients and families lost in a maze of tests, misdiagnoses, missed opportunities to participate in clinical trials, and delayed treatment. Summit 2025 brings together a diverse group of stakeholders – patient advocates, industry leaders, policymakers, clinicians, researchers and payers – to chart a collaborative path towards a future where timely and accurate diagnoses are a reality for all with rare diseases.

Save the Date: Rare Disease Day 2025 Conference
2025-04-29 08:00 - 2025-05-01 17:00 (America/Denver)

Myotonic Dystrophy Foundation Conference 2025
2025-05-01 08:00 - 2025-05-04 17:00 (America/New_York)
MDF invites you to attend the Professional Track at the 2025 MDF Conference in Indianapolis, Indiana from May 1st to May 4th! Sometimes called the “DM family reunion” – the MDF Conference is an event to unite the DM community in our journey towards treatments and a cure for myotonic dystrophy (DM). This year’s program includes educational sessions exploring research, drug development, symptom management, and other critical topics for DM families, plus opportunities for formal and informal networking, wellness, and fun!

Save the date: Redefining Patient Engagement
2025-05-08 08:00 - 17:00 (America/Denver)

Milestones & Momentum: 10 Years of Progress, 10 Years of Possibility
2025-05-21 18:00 - 20:30 (Europe/Paris)
RDI will be holding a side event at World Health Assembly 78 in Geneva. Join them as the community celebrates 10 years of progress and looks forward to 10 more years of redefining what’s possible!

CureDuchenne 2025 FUTURES National Conference
2025-05-22 08:00 - 2025-05-25 17:00 (America/Kentucky/Louisville)
This annual event is dedicated to providing education, connection, and hope to the entire Duchenne community.

European Society of Human Genetics Conference
2025-05-24 08:00 - 2025-05-27 17:00 (Europe/Vienna)

unfolding PANS PANDAS | reshaping BRAIN+BODY health
2025-05-29 08:00 - 2025-05-30 17:00 (America/Edmonton)
This inaugural national conference brings together leading experts from Canada and beyond to explore the physiological and psychological complexities of Pediatric Acute-onset Neuropsychiatric Syndrome (PANS) and Pediatric Autoimmune Neuropsychiatric Disorder Associated with Streptococcal Infections (PANDAS).

Unity Through Rheumatic Diseases: In-Person Event
2025-05-31 08:00 - 17:00 (America/New_York)
Take a Pain Check Foundation is thrilled to invite you to Unity Through Rheumatic Diseases, an in-person event dedicated to promoting collaboration and driving change in rheumatology. Join us on Saturday, May 31, 2025, from 8:00 AM to 5:00 PM EST at the InterContinental Toronto Centre (225 Front St W, Toronto, ON M5V 2X3) for a day of insightful discussions, networking, and empowerment.

Advancing Innovation in Rare Disease Research
2025-06-02 08:00 - 2025-06-03 17:00 (America/Vancouver)
Join researchers, clinicians, and industry leaders to discuss real-world research breakthroughs, novel #ClinicalTrial designs, and how NORD’s Rare Disease Centers of Excellence are leading collaborative efforts in #RareDisease innovation.

Superficial Siderosis Symposium 2025
2025-06-11 08:00 - 2025-06-13 17:00 (America/Halifax)
Join the Superficial Siderosis Research Alliance (SSRA) for a three-day symposium dedicated to patients, caregivers, clinicians, and researchers focused on advancing care, treatment, and research for superficial siderosis.

ALS Canada: Webinar-Driving Progress Forward
2025-06-18 12:30 - 14:00 (America/Edmonton)
In anticipation of Global ALS Awareness Day on June 21, join ALS Canada’s CEO Tammy Moore and the ALS Canada Team for a free, insightful webinar. Discover the comprehensive services and programs ALS Canada offers to Canadians living with and affected by ALS.
This engaging session will cover all mission areas, including community services, advocacy, research, and knowledge exchange, showcasing the extensive resources available nationwide.

Defeat Duchenne Canada-Industry Partner Update: REGENXBIO
2025-06-23 16:00 - 17:00 (America/Edmonton)
Join Defeat Duchenne Canada for an educational webinar featuring Dr. Jahannaz Dastgir, Executive Director of Clinical Development at REGENXBIO, as she shares the latest updates on RGX-202, an investigational gene therapy for the treatment of Duchenne muscular dystrophy, and their Phase 1/2/3 AFFINITY DUCHENNE® trial.
This session will provide valuable insights into the science behind RGX-202, trial progress, and what this potential investigational treatment could mean for the Duchenne community. Whether you are a parent, caregiver, clinician, or advocate, this webinar is an opportunity to stay informed and ask questions directly to the team leading this important research.

THE FESTIVAL OF GENOMICS & BIODATA Boston
2025-06-24 08:00 - 2025-06-25 17:00 (America/New_York)
The Festival is designed principally for scientists and clinicians who are working in the fields of genomics, other omics, and/or using biodata , to further research, drug discovery, healthcare and – ultimately – deliver better patient outcomes.

Topics covered include genomics, single cell/spatial biology, multiomics, biodata, cancer research, drug discovery, AI, microbiome, proteomics, liquid biopsy and so much more.

United in Hope: 2025 Global Prader-Willi Syndrome Conference
2025-06-24 08:00 - 2025-06-28 17:00 (America/Denver)
