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.

ACT Canada Webinar Series: Engaging patient partners in your CIHR grant application: Everything you ever wanted to know with Maureen Smith & Andrea Chow (Ontario)
2025-01-16 12:00 - 13:00 (America/New_York)
Learning Objectives:

Review the ways in which patients can contribute as partners on your CHIR grant application
Identify the challenges and barriers to patients in fulfilling grant application requirements and how researchers can support patient partners
Describe the considerations specific to grant application requirements for researchers working with patient partners from groups who are frequently under-served by health research

Webinar - Securing Funding for Clinical Trials
2025-01-23 12:00 - 13:00 (America/New_York)
Webinar objectives:

Identify various funding strategies for pediatric and perinatal clinical trials in Canada, including academic grants and industry partnerships.

Develop realistic expectations regarding the timeline and effort required to fund clinical trials through shared examples.

Understand different strategies when approaching industry and academic funders and identify key factors for successful proposals.

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.

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

Celebrate Rare Disease Day: Advancing Clinical Trials Together
2025-02-28 12:00 - 13:30 (Europe/Paris)
On February 28th, in recognition of Rare Disease Day, the European project DREAMS (www.dreamshorizon.eu) invites you to a thought-provoking online webinar: “Clinical Trials in Focus: Innovations and Patient Perspectives in Rare Diseases”. Join leading experts as they explore advancements in clinical trial methodologies, outcome measures, and lessons learned from research in muscular disorders.

Webinar - Initiating Clinical Trials in Canada
2025-03-05 12:00 - 13:00 (America/New_York)

ALS Clinical Trials Unboxed
2025-03-06 13:00 - 14:00 (America/Denver)
Join ALS Canada, on March 6 at 1:00 pm ET, for ALS Clinical Trials Unboxed. In this webinar, Dr. Lauren Kett, Medical Director of Clinical Development at Amylyx Pharmaceuticals will present on the Phase 1 LUMINA study, an investigational antisense oligonucleotide (ASO) targeting calpain-2 for the potential treatment of ALS.

MDA Clinical and Scientific Conference
2025-03-16 08:00 - 2025-03-19 17:00 (America/Chicago)

NMOSD in sub-Saharan Africa
2025-03-17 18:00 - 19:30 (Europe/Paris)
The experts will provide insights and answer questions about the prevalence and incidence of NMOSD, diagnostics, available treatments, immunizations, clinical trials, and more. Patients, caregivers, clinicians, researchers, and advocates from around the world are invited to attend.
Live participants will have the opportunity to ask questions directly to the experts. The webinar will be recorded and made available for replay on TSF’s YouTube channel.

Conscience Symposium on Open Drug Discovery
2025-04-07 08:00 - 2025-04-08 17:00 (America/New_York)

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.

Genome BC’s Genomics Forum 2025
2025-04-30 08:00 - 17:00 (America/Vancouver)

Medicines Reimagined: Unlocking the Potential of Existing Drugs for Patient Benefit
2025-05-07 08:00 - 2025-05-08 17:00 (Europe/Helsinki)
Join iDR25 – the international drug repurposing conference – to unlock the full potential of medicines by driving innovation for patient benefit. From cutting-edge AI approaches transforming drug discovery to exploring innovative business models and regulatory pathways for market access

Society for Clinical Trials’ 2025 Annual Meeting “Shaping the Future: The Right Questions, Robust Answers”
2025-05-18 08:00 - 2025-05-21 17:00 (America/Vancouver)

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.

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.

Webinar - Key Considerations for Including Pediatric Populations with Neurodevelopmental Conditions in Clinical Trials
2025-06-18 12:00 - 13:00 (America/Edmonton)
Speakers: Evdokia Anagnostou and Mayada Elsabbagh Title: Key considerations for including pediatric populations with neurodevelopmental conditions in clinical trials

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.

2nd Global Cell & Gene Therapy Summit
2025-07-07 08:00 - 2025-07-09 17:00 (America/New_York)

IMPaCT Webinar - An Overview of Current and Future Clinical Trials in Children: a European Perspective
2025-07-21 12:00 - 13:00 (America/Toronto)
Dr. Eva Degraeuwe (MD-PhD) is a dedicated pediatric resident and researcher in pediatric nephrology and global clinical trial optimilisation. Working in many Global networks focused on rare and low prevalance diseases, she has completed her PhD in 2024 participating and guiding over 35 clinical trials. Moreover, she has a passion for patient engagement, especially within a clinical trial setting.
Webinar Overview:
– European network setting and the role of networks
– Pathways to clinical trial optimilisation
– Patient engagement and what has been achieved

6th International Hypothalamic Hamartoma Symposium
2025-09-04 08:00 - 2025-09-05 17:00 (America/Belize)
Presented by Hope for HH and Hospital Sant Joan de Déu Barcelona, this symposium will gather leading experts, patients, and caregivers from around the world to share the latest research, clinical advances, and treatment strategies for Hypothalamic Hamartoma (HH)! Alexis Arzimanoglou and Helen Cross are part of the Symposium Committee.

Rare Trials Summit
2025-09-09 08:00 - 2025-09-10 17:00 (America/Edmonton)
Bringing together key stakeholders in the clinical research community to accelerate the development of new rare disease therapies from average 10-12 years to under 3 years.

IMPaCT Webinar: Lessons Learned from the Innovative Pediatric Clinical Trials (iPCT) Network
2025-09-10 10:00 - 11:00 (America/Edmonton)
Speakers: Tannis Erickson & Naveen Poonai Title: Lessons Learned from the Innovative Pediatric Clinical Trials (iPCT) Network

2025 NMD4C Annual Meeting
2025-09-11 08:00 - 17:00 (America/Toronto)
Registration is now open for the 2025 NMD4C Annual Meeting!
Join NMD4C in Ottawa on Thursday, September 11, for a full day of dynamic discussions, research updates, and networking opportunities—just ahead of the 7th Ottawa International Conference on Neuromuscular Disease & Biology.

Ottawa NMD 2025 Conference
2025-09-11 08:00 - 2025-09-13 17:00 (America/Toronto)
7th Ottawa International Conference on Neuromuscular Disease and Biology will take place at the Fairmont Château Laurier in Ottawa, Canada on September 11-13, 2025.
The Ottawa NMD 2025 conference will offer an outstanding program emphasizing recent breakthroughs in basic and translational research and clinical discoveries in neuromuscular disease.

Undiagnosed Hackathon 2025
2025-09-21 08:00 - 2025-09-24 17:00 (America/Chicago)
350 million people live with an undiagnosed disease worldwide. Genome sequencing can provide answers for 40% of the People Living With Undiagnosed Disease (PLWUD) but around 60% still don’t receive a diagnosis. The purpose of the Undiagnosed Hackathon is trying to find new ways and collaborations to solve the undiagnosed diseases, that can’t be solved today. In this unique event multidisciplinary teams of clinicians, bioinformaticians/molecular biologists, scientists, developers, AI specialists and brilliant minds will work together to tackle the most difficult diseases.

6th Western Canadian Neuromuscular Conference
2025-09-25 08:00 - 2025-09-27 17:00 (America/Edmonton)
The 6th Western Canadian Neuromuscular
Conference is confirmed for 2026! Don’t miss
the opportunity to network with leading experts
and stay at the forefront of medical
advancements in the field of neuromuscular
diseases.

Canadian Rare Disease Symposium: Preparing for Clinical Trials – International Regulatory Insights, and cGMP Gene Therapy Manufacturing capabilities in Canada
2025-10-01 13:00 - 16:00 (America/Toronto)
The Stem Cell Network (SCN) and the National Research Council Canada (NRC) are excited to contribute to the national conversation on cell and gene therapy for rare diseases. Building on the 2024 event titled “Canadian Rare Disease Ecosystem Symposium: Genetic Therapies for Rare Disease”, SCN and NRC are pleased to invite you to the Canadian Rare Disease Symposium: Preparing for Clinical Trials – International Regulatory Insights, and cGMP Gene Therapy Manufacturing Capabilities in Canada–a focused half-day event dedicated to advancing gene and cell therapy development in Canada through regulatory alignment and manufacturing readiness.
This virtual symposium will convene international regulatory authorities, Canadian manufacturing experts, and rare disease developers to explore two critical enablers of translational success: clinical trial readiness and cGMP manufacturing capacity.
