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VIDEO: This ‘Bootcamp’ Helps Parents Advance Rare Disease Research to Save their Children

More than 100 nonprofit patient organizations have participated in Ultragenyx-led Rare Bootcamp events since 2017

We call it ‘Bootcamp’ because you are warriors, and this is a battle.

“We call it ‘Bootcamp’ because you are warriors, and this is a battle. You will need endurance, you’ll need strength.” With these words, our founder and CEO Dr. Emil Kakkis kicked off the three-day Rare Bootcamp, where a group of patient advocates and parents who are actively pursuing potential rare disease treatments learn directly from experts how to advance the research and development of new medicines. Dr. Kakkis, a medical geneticist by training, is speaking from direct experience – he started his career in rare disease drug development when the Dant family, desperate for a treatment, came to his academic lab and funded the research that ultimately led to a first-of-its-kind, lifesaving medicine for their son, Ryan.

Photo: Emil D. Kakkis, M.D., Ph.D. speaks at a Rare Bootcamp event in 2022

Since then, Dr. Kakkis founded Ultragenyx to identify and develop transformative therapies for rare genetic diseases. Because Dr. Kakkis and the Ultragenyx team understand how difficult the path of drug development can be for small and often family-run organizations, each year we bring together a group of like-minded industry and academic experts to share our knowledge and expertise and help the entire rare disease community move forward.

I think it’s a failure of the system that people have to try to develop drugs to save their kids.

“I think it’s a failure of the system that people have to try to develop drugs to save their kids,” says Dr. Kakkis. “But when their child is at risk, these parents find the strength to become the warrior and show up here.” At Ultragenyx, we’re committed to sharing our science and expertise to support them on their journey.

Photo: Rare Bootcamp participants talk during an event in 2022

The first Rare Bootcamp took place in 2017, and each year the event offers us the chance to connect with parents and patient advocates who inspire us and the other passionate presenters and sponsors. Due to overwhelming demand, we held two different Bootcamp events in 2022, which brought dozens more warriors into the Rare Bootcamp community.

Here are some of the key takeaways from participants and sponsors of the 2022 Bootcamp events, in their own words:

If you’re a parent of a child with a rare disease, there’s a lot of work to be done. ​At the same time there’s no reason ​to be disheartened; there is a future for your child.​

Jainu Jogani
Hope4Harper CDKL5

This is the most important event I attend all year and the one that means the most to me. The participants are all heroes – they’ve taken on a second and third full-time job, becoming experts in a rare disease. They show up here with such positivity, openness, and commitment.

Bruce Bloom
Rare Bootcamp speaker and
Chief Collaboration Officer at Healx

Bruce Bloom

Jennifer Sills

I went to Rare Bootcamp to learn and make connections in the hope that it would help accelerate our research program, but I came away with much more. I came away with strong partners and friends who are in the trenches with me on this journey.

Jennifer Sills
CSNK2A1 Foundation

I was blown away by how open and supportive everyone at the Bootcamp was – participants and organizers alike. I feel like I came away with a group of people in my corner for questions and advice going forward. Now it’s like I actually have some ‘coworkers’ even if we’re all working on different diseases.

Hannah Lowe
L-CMD Research Foundation

The Rare Bootcamp events are a natural outcome of our patient-focused culture here at Ultragenyx. The entire company is focused on our mission to treat as many rare disease patients as possible with the utmost urgency, and we believe that sharing our knowledge and supporting others on their journey can accelerate the timeline of rare disease drug development for everyone.

More than 100 parent and advocacy organizations have now participated in our Rare Bootcamp events

We’re proud to have recently reached a milestone – more than 100 parent and advocacy organizations have now participated in our Rare Bootcamp events, and these warriors are making an impact. Some of our participating organizations now have clinical studies underway, others are in the process of identifying a potential therapeutic approach, and all of them now have a strong community of fellow parents, biotech experts, and academic researchers in their corner.

Photo: Arjun Natesan at a Rare Bootcamp event in 2022

I want to thank the Ultragenyx team members and leaders who support the Rare Bootcamp events and generously share their time and expertise, our speakers and sponsors for being our Bootcamp “faculty” and making the event possible, and most of all the rare disease parents and advocates who attend the Bootcamp events – you continue to inspire us to keep going beyond, every day.

Arjun Natesan is vice president of translational research at Ultragenyx