RP-A501 is being developed for Danon Disease, a rare genetic disorder characterized by thickening and weakening of the heart muscle, often resulting in heart failure, and for male patients, frequent death during adolescence or early adulthood. Rocket is the first company demonstrating safety and efficacy data in clinical studies for gene therapy targeting the heart.

An open-label, global Phase 2 pivotal clinical trial of RP-A501 for the treatment of Danon Disease has commenced. The trial is expected to enroll 12 male patients from the U.S. and EU. The trial will assess a co-primary endpoint consisting of LAMP2 protein expression and left ventricular (LV) mass reduction from baseline at 12 months.

Phase 2 Trial Sites

For more details about the RP-A501 for Danon Disease clinical trial, visit clinicaltrials.gov.

Frequently Asked Questions

The Natural History of a Disease is the typical progression of the disease over a person’s lifetime starting from its onset

Natural History studies are observational studies that gather comprehensive data on the course of the disease to gain a better understanding of its progression and impact on patients. Data captured may include information on the physical signs and symptoms patients experience, test results (for example blood tests or imaging), genetic information, and health-related quality of life, over a period of time. This information helps describe the disease course and identify important factors (e.g. demographic, genetic, and/or lab test results), that may contribute to better or worse disease outcomes.

Travel, accommodation, and food stipend would be provided. Study assessments are provided free of charge.

Natural History Studies are particularly important for rare diseases. Given the diversity of symptoms and the small size of the patient population, these studies are often the only opportunity for researchers and physicians to address significant knowledge gaps and obtain a robust understanding of the disease and patient experience.

Engaging in a Natural History Study offers numerous benefits to the community, such as:

  • Raising awareness in medical, academic and pharmaceutical sectors
  • Empowering rare disease patient communities with knowledge and support for advocating better healthcare policies and support services
  • Enhancing patient care with improved insights into disease progression, allowing for more personalized treatment plans and potentially earlier detection of complications
  • Contributing to advancements in healthcare

These studies provide various avenues to help inform and design clinical trials, such as:

  • Highlighting areas of unmet medical need
  • Understanding characteristics of the patient population to define a treatment benefit from a patient’s perspective.
  • Describing disease progression, enabling the identification of clinical outcome measures and biomarkers
  • Provide patient-centric data for health authorities such as the FDA, and other public health institutions to use in their review processes of investigational treatments.
Learn more about Rocket’s clinical trial for FA

Interested in learning more?
Visit our Patients & Caregivers page or email us at clinicaltrials@nullrocketpharma.com

View more details about the Phase 2 pivotal trial of RP-A501 and natural history study on clinicaltrials.gov.

To read our Expanded Access statement, click here.

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:

Fanconi Anemia (FA)

Leukocyte Adhesion Deficiency-I (LAD-I)

Pyruvate Kinase Deficiency (PKD)

Danon Disease

Plakophilin-2 Related Arrhythmogenic Cardiomyopathy (PKP2-ACM)

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 [email protected].

Fanconi Anemia (FA) Resources

Leukocyte Adhesion Deficiency-I (LAD-I) Resources

Danon Disease Resources

Pyruvate Kinase Deficiency (PKD) Resources

Plakophilin-2 Related Arrhythmogenic Cardiomyopathy (PKP2-ACM) Resources

Rare Disease Resources

Genetic Testing

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

Learn more about our commitments

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!

Click here to submit

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.

Click here to submit
Video Icon

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.
Click here to submit

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.
[email protected]

Expanded Access Policy

To read our Expanded Access statement, click here.

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