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Scientific, reliable information for patients and the public
View all sessions from this spring 2025 inaugural event! Save the date for the next Summit, March 11-12, 2026.
Learn more about how gene therapy works for specific diseases.
Learn about approved treatments for blood cancers, such as leukemia and lymphoma.
Learn about this rare genetic disorder that causes harmful buildup of glycolipids throughout the body and how gene therapy may be able to address its symptoms.
These are a set of rare genetic disorders including cerebral adrenoleukodystrophy (CALD) and metachromatic leukodystrophy (MLD).
Stay up to date on news and events relevant to patients, caregivers, advocates, and anyone else affected by rare diseases. Sign up to get each issue delivered straight to your inbox every other month!
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.
We are thrilled to announce that ASGCT has been named a winner of the 2021 "Power of A" Silver Award from the American Society of Association Executives (ASAE) for our Patient Education Program!