The information contained in this website is intended for healthcare providers and not for the general public.
GeneReviews CTX: The National Institutes of Health maintains GeneReviews—expert-authored, peer-reviewed disease descriptions ("chapters") presented in a standardized format and focused on clinically relevant and medically actionable information on the diagnosis, management, and genetic counseling of patients and families with genetic conditions, including CTX.
Genetic testing registry (GTR): The National Institutes of Health maintains a database of available tests for CTX and other genetic conditions.
National Organization for Rare Disorders (NORD) CTX: NORD is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them.
National Institutes of Health – Genetic and Rare Diseases Information Center CTX: Find additional information on CTX on this page, hosted by the US Department of Health and Human Services.
About CTX.com/patient: See the patient/caregiver section of this site for resources and information tailored to those who have a family member with a possible or confirmed diagnosis of CTX.
United Leukodystrophy Foundation: CTX is sometimes characterized as a leukodystrophy. The United Leukodystrophy Foundation maintains a CTX page and has a discussion forum where patients with CTX and their families can connect and offer support.
Links to external sites are provided for informational purposes only and Mirum Pharmaceuticals, Inc. does not endorse or guarantee in any way the services or advice provided by them.