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Showing articles from rare disease tag

Waste Not, Want Not – Making the Most of Small Patient Population Data

_***Recommended for Young Investigators/Researchers**_ **Strategies for Successful Research in Small Patient Populations** * Discuss how to support and engage in research activities and develop interest for small patient population studies **Understanding Measurement Tools to Capture Patient Data in Rare and Ultr…

Equity & Ethics in Context – Raising the Bar for Access in Rare Disease

**The Rare Disease Medical Ethics Landscape** * Opportunities and challenges in informed consent for investigational therapies and emerging science * Cultural competency in patient education and disclosures as a tool to build trust in research activities * Inclusivity & Representation in the Context of Small Pa…

Global Genes LIVE!: From Data to Decisions - Leveraging Genetics Data Beyond a Diagnosis

Will genomic sequencing or genetic testing give you and your family the answers you seek? And what happens when the results come in? In this discussion, learn what types of testing are available for rare diseases and what data or diagnoses they may show. Hear stories from families who have gone through testing and how…

Global Genes LIVE!: Gene to Therapy: How Individualized Therapies Are Becoming Reality

Personalized medicine is revolutionizing patient care as we know it. Hear from our panelists as they share case studies of the progress enabled by personalized medicine. They will share the risks and opportunities new technologies have created in this space as well as the hope personalized medicine brings for the futu…

NTM Lung Disease Webinar

Coughing, fatigue, or shortness of breath that won't go away? In partnership with INSMED, we dive into the deeper risk factors, diagnosis, and testing for rare lung diseases during this webinar. Learn from leading specialist Dr. Patrick Flume and patient and advocate Debbie B. about the management of this condition an…

Global Genes LIVE!: Navigating the Transition of Care for Young Adult Patients

Hear firsthand from young adult rare disease patients who have gone through the transition from pediatric to adult care. They discuss approaches you can use for yourself or a patient you care for to help make the transition of care coordination from parent or guardian to child more smooth. They also help identify the …

RARE Drug Development Symposium: Exploring the World of Funding Research

Expanding your network, building the credibility of your organization, and speaking on behalf of the patient are all crucial steps in gaining and sustaining funding for your research. Hear how you can become a connector for people in your rare disease community and creative ways to fund research, from featured speaker…

2019 NEXT: Imagining the Future of Rare Disease

![][1]The pace of innovation continues to accelerate, thanks in no small measure to the growing sophistication of rare disease patients and their families. In _NEXT: Imagining the Future of Rare Disease_, we take a wide view of the rare disease landscape to look at how technology is improving the ability to understand…

Global Genes LIVE! Be Heard: Patient Perspectives in Novel Therapeutics Value-Based Discussions

What are the biggest challenges to getting your perspectives included in value-based discussions around novel therapeutics? Hear from advocates who are participating in discussions on value demonstration and reimbursement models for novel therapeutics and learn to leverage your patient perspective to provide a more ho…

RARE Drug Development Symposium: How to Be a Great Research Partner

Collaboration is crucial when it comes to developing drug therapies for rare diseases. Nan Doyle, Adam Sherman, and Onno Faber discuss how a patient is able to be involved in every step of the process, and what pharmaceutical companies and researchers can do to form trust in these collaborative partnerships.

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