Patients & Families

Having a loved one or living oneself with a serious genetic disorder is a tribulation that many people can scarcely fathom. It requires courage and endurance to make it through each day, and to investigate and understand various medical therapies. Although some treatments have been effective, many definitive treatment options (including allogeneic stem cell transplant) involve risks and may cause additional lifelong complications.

Rocket Pharma was founded with the goal of creating therapies that provide a corrected (healthy) gene into a patient’s own stem cells, with the intent of markedly reducing or even eliminating the devastating and life-threatening consequences of diseases such as Fanconi Anemia, Pyruvate Kinase Deficiency and Leukocyte Adhesion Deficiency.

We recognize that for many patients and families, this cannot happen fast enough. We also realize that any treatment, no matter how carefully designed, can involve risks and must be studied carefully, in compliance with health authority and hospital regulations and with highest ethical standards, with the goal of minimizing potential harm.

Our challenge is working with the right balance – moving forward with the speed and urgency that these diseases demand, while making sure that no one is put unnecessarily in harm’s way. Our challenge is to enable patients to receive potentially curative gene therapy while also collecting and studying the detailed information required for clinical trials and for health authorities such as the US Food and Drug Administration or European Medicines Agency. We cannot promise that every laboratory experiment will work as hoped for.

We cannot promise that every patient with a rare disorder will be eligible for a clinical trial, yet. We cannot promise that every patient who receives treatment in a clinical trial will have successful reversal of their disorder.

We can promise that each step leading up to our clinical trials, and every decision made for our trials will be performed both with utmost urgency and appropriate caution. We can promise that your voices will be heard, and that the needs and hopes of patients, families, medical caregivers and research experts will be ingrained in our programs. We can promise that our plans will be communicated, and that regardless of early success or failure, we will continue to seek better ways of treating these diseases until our goals our achieved and patients with rare and devastating diseases can receive highly safe and effective gene replacement therapies.

Even though they are rare, diseases like Fanconi Anemia, Pyruvate Kinase Deficiency and Leukocyte Adhesion Deficiency have caused extensive suffering and shortened many lives. Together, with the help of patients, families, support groups, medical professionals, research experts and our partner organizations, we believe we can make a world of difference.

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