Patients & Caregivers

Children Smiling Patients and Caregivers Hero

Patients with rare diseases are our North Star. Our constant reminder that keeps us centered on our path to seeking cures through gene therapy. Only by understanding their journey can we move forward in ours.

Living with a rare disease is much more than just a physical fight. It involves fighting misconceptions, finding informed and compassionate caregivers, and seeking accurate information about available treatment options. Rocket is an ardent advocate for the rare disease community. By committing to raising awareness and contributing to public education on a higher level, we’re also aiming to ensure patients and their loved ones gain access to information and resources that will help connect and empower them.

Clinical Trials

We are purposefully focused on patient communities and conditions with devastating, life-long effects, and little to no treatment options, studying ways to correct the disease at the genetic level to enable people to live long and full lives. The diseases we are pursuing in ongoing clinical studies include:

Educational Resources

We encourage you to explore more about the following organizations that offer resources and information for patients and caregivers. If your group would like to be included in our list of patient resources, please reach out to us at

Genetic Testing

Rocket is partnering with some of today’s leading minds to help patients with rare diseases pursue an early and accurate diagnosis.

Rocket patient and Caregivers Our Events Pictures

Our Events

Rocket hosts several patient-focused events during the year, including our annual Rare Disease Day event open to patients and families as well as members of the biotech and academic communities.

Check out photos, videos and more from our Rare Disease Day events at the links below or visit our YouTube page.

Hear Rare Roar Project

As part of Rocket’s efforts to raise awareness for rare diseases, especially Fanconi Anemia, Danon Disease, Leukocyte Adhesion Deficiency-I and Pyruvate Kinase Deficiency, we have launched the Hear Rare Roar Project. The purpose of this project is to bring important and impactful stories about patients’ and families’ experiences with their rare disease to the forefront and spread awareness of these diseases. Interested in participating and being featured on Rocket’s website? Read more below!

Patient and Family Photo Crowdsourcing

Interested in having your photo featured on our website and on social media? Submit your favorite photo, along with a note about why your photo perfectly captures your experience with your rare disease. Be creative!

Patient and Family Story Submissions

Not sure about having your photo featured but interested in sharing your story? Write a letter about your experience to help others understand how it feels to live with a rare disease. This can be addressed to your disease, to the team at Rocket or to the broader community. Whatever feels most comfortable for you. Letters will be shared on Rocket’s social media channels.

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Patient and Family Video Submissions

Interested in sharing your story via video? Record a 1-4 minute long video of you talking about your experience. Videos will be featured on Rocket’s social media channels.

Contact Us

If you are interested in learning more about any of our clinical programs or patient and family events, please contact our Patient Advocacy team:

Patient Advocacy
Rocket Pharmaceuticals, Inc.

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