Providing education, support, advocacy, and promoting research
for CTX patients, their families, and medical professionals
who treat and study this rare disease.
The CTXA is working diligently as the engine to drive the important work required to meet the unmet needs of CTX patients. There are four key initiatives:
Education focused on earlier diagnosis and advocacy efforts for newborn screening for earlier detection to improve outcomes and therapeutic intervention.
Advocate for formal FDA indication and approval of the current treatment, which is being provided off-label and not guaranteed to be accessible in the future.
Build a community of CTX patients and families who focus on the unique and specific patient needs and represent CTX with a larger, collective voice for research and improved therapies.
Be a resource for physicians, allied healthcare providers, and other caregivers for medically vetted information on CTX, including the potential of providing laboratory testing facilities and resources on how to obtain medication.
Short-term goals include:
Mid-term goals include:
Long-term goals include:
For more information or questions, please contact Jean Pickford at firstname.lastname@example.org.
Join our mailing list to receive information and news as we begin to gather and expand the CTX community.
1586 Sumneytown Pike
PO Box 1322
Kulpsville, PA 19443