Past Events

September 17-18th, 2024 | Pennsylvania Convention Center, Philadelphia

Closing Keynote Panel

Rare Disease Day 2024

SYNGAP1: Birth of a Legacy

Nova Scotia, Canada

May 24, 2024

The Corinthians 93, Mason Lodge

March 27, 2024

Trends and Strategies in Integrating the Patient Voice

Case Study: Creating and Designing Meaningful Patient-Centered Translational Research

Patient-Focused Drug Development Meeting Report Overview

Keynote Panel Discussion: Expectations and Future Direction for Cell and Gene Therapies

Presentation: Reverse Engineering for Rare Disease Drug Development

Real-World Data for Rare Diseases: "A Boy and His Dog"

Patient Perspectives on Gene Therapy: Oral Testimony and Presentation

Death By Zip Code: The Newborn Screen Crisis in the United States

Exploring the Patient Advocates' Role in Reducing Access Barriers: The Call For Health Equity

Listening Session

Reverse Engineering of Drug Development

Co-Moderator: Tools and Resources for Nonprofits

The role of innovation in clinical pharmacology can play in patients' lives, and the role patients can play in advancing science.

Reverse Engineering for Drug Development in Rare Diseases

Shaping Healthcare Policy, Inserting the Rare Disease Voice

Topic: Getting Involved in Rare Disease Research

Reverse Engineering for Rare Disease Drug Development

Chairwoman: Breakout Sessions

Panel: A Look at Rare Disease Listening Sessions and PFDDs with NORD and FDA

Panel: Patient Data Ownership - Collection, Stewardship, & Lessons Learned from Registries

Engaging Online Communitues and Influencers

Reverse Engineering for Rare Disease Drug Development

Keynote speaker at the first US National CBD Summit in Washington, DC, was an honor.

Panel on Clinical Trials: Precision Approaches to Rare and Not So Rare Disorders with Breakout Discussion

From Competition to Collaboration — Improving Industry Habits for Funding and Sustaining Registries

Topic: “Germline gene editing: A patient perspective.”

Topic: The Challenges Around Living and Coping with COVID-19 and Special Needs Populations

Moderator: Future of Healthcare Policy and Regulation in the Digital Age

Building Sustainable Business Strategy: Partnering with Academia and Industry

Moderator: We discussed challenges in accessing patient communities, mental health needs, strategic ways to engage with patient communities, and the challenges around splinter groups and educating the general population, including rural and diverse populations.

Discussing Cell and Gene Therapy for Rare Diseases and Big Data.

Teaching the importance of Advocacy and Research.

Real World Data For Rare Diseases: How to make drug development more feasible for Rare Diseases and improve regulatory decision making.

DAdvocacy in Action: The Perspective of an Academic Neurologist on the Hill.escription goes here

I shared Beckett's story with 900+ health occupation students about the different opportunities available in the health field, including legislative and data technology.

Optimize Reimbursement and Access Through Proactive Planning in Clinical Development

CASE STUDY: Bringing Data Back to Patients — Empowering Patients through Data Sharing

Moderator: Discussion on Economic Burden of Rare Diseases