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Showing articles from Drug development tag

The Future of Rare Metabolic Syndromes

Decades of research are yielding new, innovative therapies for rare metabolic syndromes. Learn about the potential for gene therapy, peptide therapeutics, and more. Speakers: James Doyle, PhD, Russell Gotschall and Yael Weiss, MD, MSc. 

Patients and Caregivers: Shaping Clinical Drug Development

Starting a clinical trial is no simple task. From developing a protocol to patient recruitment to U.S. Food and Drug Administration (FDA) submission, there are a wide array of complex and closely monitored processes along the way. Patient and community involvement help ensure study questions are relevant and that the …

Global Genes LIVE!: Mapping Patient Inclusion in Drug Development

Are you in the early stages of learning how to engage in the drug development process as an organization or individual patient? Then join us to get a strong understanding of the drug discovery and preclinical development processes. We will discuss the lifecycle of traditional therapies and new trends in research and d…

RARE Drug Development Symposium: Strategies for Small Patient Populations

Learn how to leverage your Small Patient Population's specific advantages to enact flexibility in rare disease regulation. While research for small patient populations presents its own challenges, Dr. Tricia Gooljarsingh, Dr. Matt Might, and James Valentine, all address strategic ways to address them.

RARE Drug Development Symposium: Improving Clinical Transparency & Data Sharing

It’s well known that data sharing and collaboration when advancing rare disease research is beneficial to all. TJ Sharpe and Jenn McNary discuss how patient populations, organizations, and industry members can better create an environment of transparency.

RARE Drug Development Symposium: Cell Models in Rare Disease Research

**Day 1: **Optimizing the use of cell models in rare disease research can sound daunting. Dr. Deborah Requesens and Herve Tiriac discuss how cell models can be used to set a strong foundation for your research and encourage diversity in clinical trials. **Day 2: ** The value of cell models lies in setting a good fou…

RARE Drug Development Symposium: Shifting Landscapes in Rare Disease Research

Dr. James Wilson outlines how we can use our own history to create usable research data at the 2020 RARE Drug Development Symposium. Learn how logging and compilation of natural history data holds an opportunity to transform the future of rare disease research.

RARE Drug Development Symposium: Galvanizing Next Steps in Rare Disease Research

What silver linings can we create for rare diseases from the COVID-19 pandemic? Panelists Dr. David Fajgenbaum, Dr. Jeremy Levin, and Dr. Katherine Beaverson discuss the future of rare disease research and why working together holds our best opportunity for hope.

Early Drug Development What Patients Bring

The drug development process is often lengthy and requires many resources. In this session, receive a comprehensive overview of the early stages of the drug development process and learn how advocates can help develop these needed resources and push their communities forward. Speakers: Laura King Edwards, Steven Gray…

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