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.

RARE DISEASE DAY 2025

RARE DISEASE DAY 2025

Rocket Pharma - Rare Disease Day Logo
Rare disease day 2025

ROCKET PHARMA CELEBRATES RARE DISEASE DAY 2025

EVERY STRIPE TELLS A STORY

Approximately 10% of Americans are affected by a rare disease, with over 300 million people impacted globally. Many individuals and families facing these conditions struggle with isolation and unanswered medical questions –but together, we can change that. Rare Disease Day is a global movement to raise awareness and support for those living with rare diseases.

This year, Rocket Pharmaceuticals proudly hosted Rare Disease Day 2025: Every Stripe Tells a Story at the Park Chateau in East Brunswick, NJ.

Every Stripe Tells a Story brought together the patient community, patient advocates, genetic counselors, industry leaders, regulatory and access experts, and Rocket employees to discuss key topics such as Danon disease, making impactful memories, the importance of genetic testing, and innovations shaping the future of rare disease therapies.

Welcome | The Meaning of Rare Disease Day

Kinnari Patel, PharmD, MBA, President, Head of R&D and Chief Operating Officer

Danon Disease | Patient, Family, and Advocate Perspectives

Brittany & Micah, Living with Danon disease

Jenny Hsieh, Co-Founder and President, Danon Foundation

Amy Atkinson, Co-Founder, Danon Foundation

Latika Hickey, PharmD, Senior Director, Patient Advocacy, Rocket Pharma

Advocacy Spotlight | RTW Foundation

Joe Katakowski, PhD, Director, Research, RTW Foundation

Making Memories that Matter

Jennifer Bennett, Assistant Vice President of Mission Delivery, Make-A-Wish Foundation of New Jersey

Dominique Gleason, Director of Events, Make-A-Wish Foundation of New Jersey

Fanconi Anemia | Voices of Hope

Erika Zannou-Mantri, PharmD, PhD, MBA, SVP, Global Program Head & Development Operations

Breaking Barriers: Pathways to Support Innovation and Access for Rare Disease Therapies

Michael Lehmicke, Senior VP, Science and Industry Affairs, ARM

Alan Minsk, Partner, Arnall Golden Gregory

Mike Cibulsky, AVP, Head of U.S. Value, Access & Trade, Rocket Pharma

From DNA to Diagnosis | The Transformative Power of Genetic Testing in Rare Disease

Cara Barnett, MS, CGC, NSGC Cardiovascular Genetics Expert

Xiao P. Peng, MD PhD, Director of Advanced Diagnostics, New York Center for Rare Diseases at Montefiore Einstein

Monisha Sebastin, MS, CGC, The Children’s Hospital at Montefiore

Tom Faria, PharmD, Vice President, Head of Global Medical Affairs, Rocket Pharma

Closing Remarks

Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma

For any questions, please reach out to [email protected]

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