Episode 6: Wes Michael | Building the Infrastructure for Patient Voice

Wes Michael brings a systems-level perspective to the rare disease ecosystem through his work as the founder of Rare Patient Voice. With a background in market research, Wes recognized early on that healthcare organizations lacked ethical and effective ways to listen to patients, especially those navigating rare and complex conditions.

In this episode, Wes shares how Rare Patient Voice was created to connect researchers, companies, and institutions with patients and caregivers in ways that respect their time, compensate their expertise, and avoid exploitation. We discuss why patient voice must be built into research and development early, how compensation changes power dynamics, and what happens when listening becomes a foundational part of decision-making rather than an afterthought.

This conversation adds an important infrastructure layer to the Rare Rebels™ map, showing how patient voice moves through systems, how it becomes usable, and why ethical listening is essential to progress across rare disease research, trials, and care.

Guest: Wes Michael — Founder of Rare Patient Voice, creating ethical, scalable infrastructure that enables patients and caregivers to share their experiences and shape research, development, and healthcare decisions.

About Rare Rebels™ Rare Rebels™ is an ongoing effort to map the rare disease ecosystem through the people leading the charge, documenting how change happens and how connections across science, advocacy, infrastructure, and lived experience shape what becomes possible.

Episode 5: Jennifer Siedman | Making Space for Care, Grief, and Hope

Jennifer Siedman brings a steady, thoughtful presence to conversations that many families struggle to name. As a parent and leader at Courageous Parents Network, her work focuses on helping families navigate serious illness with clarity, compassion, and support, especially when there are no easy answers.

In this episode, Jennifer reflects on how her personal journey informs the topics Courageous Parents Network engages with, including different types of grief, the evolving meaning of hope, and the emotional complexity of decision-making in medical care. We talk about how families live with uncertainty over time, and why support must extend beyond moments of diagnosis or crisis.

This conversation adds an important layer to the Rare Rebels™ map by centering care that is relational, ongoing, and deeply human. It highlights the role of guidance, language, and presence in helping families feel less alone as they navigate medical complexity and the long middle spaces of rare disease.

Guest: Jennifer Siedman — Parent and nonprofit leader at Courageous Parents Network, equipping families of medically complex children with language, guidance, and support to navigate care, grief, and decision-making.

About Rare Rebels™ Rare Rebels™ is an ongoing effort to map the rare disease ecosystem through the people leading the charge, documenting how change happens and how connections across science, advocacy, and lived experience shape what becomes possible.

Episode 3: Susannah Fox | Mapping the People Who Move Systems

Susannah Fox brings a unique ability to listen deeply and reflect patterns back to the people living them. In this conversation, she shares how that way of listening has shaped her work as a researcher and author, and why it matters so much in spaces where systems are fragmented and people are often left to navigate on their own.

The episode centers on Susannah’s book Rebel Health, which outlines the roles needed in a patient-led revolution and offers a framework for understanding how change actually happens. Together, we explore how those roles show up across rare disease communities, where seekers, solvers, networkers, and champions often emerge out of necessity rather than intention.

We spend significant time discussing Champions in particular, especially those who may not have a direct personal connection to rare disease. Susannah shares insights into how these individuals can play a meaningful role when they listen well, show up consistently, and align themselves with the needs of the community rather than their own assumptions.

This conversation widens the lens of the Rare Rebels™ map, showing how movements grow through connection, curiosity, and people willing to stand alongside others, even when the challenges are not their own.

Guest: Susannah Fox — Researcher, author, and patient-experience champion whose work has illuminated how communities lead innovation through shared knowledge, connection, and self-organized care.

About Rare Rebels™ Rare Rebels™ is an ongoing effort to map the rare disease ecosystem through the people leading the charge, documenting how change happens and how connections across science, advocacy, and lived experience shape what becomes possible.

Episode 2: Emil Kakkis | When Science Becomes Human

Emil Kakkis began his career wanting to focus on the science. He imagined a life in the lab, driven by research questions and discovery. What he didn’t anticipate was how deeply personal rare disease work would become.

In this conversation, Emil shares how his relationship with families affected by MPS I, particularly his partnership with Mark Dant and his son Ryan, reshaped his understanding of responsibility in science. Developing a treatment was no longer just about proving something could work. It was about staying with that work when the system made it difficult, and when walking away would mean leaving real people behind.

The episode also explores Emil’s decision to operate within the pharmaceutical industry, not as an endpoint, but as a vehicle for building strong teams and delivering high-quality therapies. Emil reflects on the challenges of moving science into practice, the role families play in shaping progress, and his broader vision for the future of rare disease, including new models that rethink how care, research, and treatment development could be structured.

This conversation reveals how relationships turn science into action, and how rare disease progress is often driven by people who never planned to lead, but chose not to walk away.

Guest: Emil Kakkis — Medical geneticist and rare disease drug pioneer who advanced enzyme replacement therapy for MPS I (Aldurazyme) and went on to found Ultragenyx and the EveryLife Foundation to accelerate rare disease treatments.

About Rare Rebels™ Rare Rebels™ is an ongoing effort to map the rare disease ecosystem through the people leading the charge, documenting how change happens and how connections across science, advocacy, and lived experience shape what becomes possible.