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Showing articles from RDDS tag

Supporting Patients, Families and Caregivers Across the Research Ecosystem and Closing Remarks

**Beyond Enrollment – Understanding Lived Experiences from Patients & Families Participating in Research** * The impacts and burden of care for patients, families, and caregivers and what researchers might not see * Implications for mental health and emotional well-being * Bridging gaps between the researchers/…

Critical Considerations Collaborating with Patients/Patient Advocacy Groups

_***Recommended for Young Investigators/Researchers**_ **Understanding Partnerships with Patients, Caregivers and Advocates in Rare Disease Research** * What are the ways in which patients and advocates work differently and/or more closely with researchers? * How can you best work with a community or patient pop…

Understanding Basket/Umbrella Trials & Master Protocols in the Context of Patient-Centered Research

_***Recommended for Patients/Advocates**_ **Defining Basket Trials, Umbrella Trials, & Master Protocols for Clinical Trials** * Defining key terminology in study design and adaptive clinical trials * Design and structure of study types Opportunities & Challenges in Basket and Umbrella Trials * Applicability of…

Cures Within Reach’s 2021 CureAccelerator Live! for Rare Diseases event

Global Genes’ partner Cures Within Reach held its philanthropic pitch event featuring 4 clinical repurposing trials for pediatric rare diseases from DEBRA Chile, Stanford University, University of Texas MD Anderson Cancer Center and University of Utah Health. This event was featured during our RARE Drug Development Sy…

Preclinical Modeling – What Advocates Need to Know about Animal & Cell Models

__*Recommened for Patients/Advocates__ **Advances in Animal Models** * How have approaches to animal models evolved in rare disease research and explore what advocates need to know to evaluate animal models in their own disease state/community? * What are animal models used for and what are the most common anima…

Data Strategy for Research Development – What Do Advocates Need to Know

_***Recommended for Patients/Advocates**_ **The Role of Patient Data from Basic Research to Approval and Access** * Understanding the role of patient data from preclinical to approval to reimbursements - the importance of planning ahead * Differentiating study/non study data Evolutions in Natural History Studies…

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…

Deep Dive – Contextualizing Endpoints and Therapeutic Advances in Rare Disease

**Understanding the Landscape for Rare Disease Endpoints** * New developments/approaches & trends * Regulatory openness to complex and innovative endpoints **Measuring Burden of Illness and Quality of Life: Aligning What’s Meaningful Between Patients & Researchers** * Opportunities to improve identification o…

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