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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.
New content coming soon!