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Helpful information to guide you through the challenging journey from diagnosis to pursuing a gene therapy.
Genetic testing, in a variety of forms, is one way to identify rare genetic diseases.
Here are information and resources to take some first steps after you've received a confirmed diagnosis of a rare genetic disorder.
Patients and advocates play an important role to initiate gene therapy research.
Here's a glimpse into using the clinical trials finder and what to expect when entering a clinical trial.
This page addresses key ethical questions that patients, their families, and the public might have about this technology.
Learn about some of the signs that might help you decide whether a provider of cell therapies can be trusted.