Precision Medicine & Rare Disease

If you work in rare diseases or rare disorders, this conference is one you cannot miss. Come and hear talks and discussions by leaders in academia, biotech, pharmaceuticals, diagnostics, clinical research, informatics, healthcare and regulatory bodies at the world’s most informative virtual conference.

Patients living with rare diseases are often persistently misdiagnosed or undiagnosed, potentially resulting in no treatment at all or treatments that are ineffective or unsafe.  Due to the lack of definitive diagnostics or efficacious and safe therapies, foundations and research centers have been raising funds for re-purposing existing therapeutics or developing new drugs (typically classified with an orphan drug status).  As the development of biomarkers and genetic signatures continues to progress, techniques and technologies are anticipated to become more precise and comprehensive, thereby reducing the time to rare disease diagnoses.

This conference will cover the following topics and more:

• Experiences from all parties – patient journeys, doctors’ consultations, payer perspectives
• Recruiting rare disease patients for clinical trials
• Tools that allow online access for patients, doctors, data sets, multi-participant meetings
• Challenges of treating rare disease patients and families during a pandemic
• How large, medium, and small companies in pharma, clinical, and diagnostic groups are addressing rare disease challenges, including translational medicine, data analysis, economic modeling, real-world data capture