The United Leukodystrophy Foundation (ULF) in the United States, in partnership with ELA International, organizes the Unlock CTX virtual meeting focused on the leukodystrophy cerebrotendinous xanthomatosis CTX, under the US Food and Drug Administration’s (FDA) Patient-Focus Drug Development (PFDD) program.
It will take place on September 14, 2021 in the form of a half-day vitual meeting which will enable patients and caregivers to participate from anywhere in the world without having to travel.
The CTX disease is an ultra-rare disease that affect the body’s ability to metabolize fats known as cholesterols, with increasing severity and impact on daily functioning.
The long delay in diagnosis and lost opportunity for treatment can have tragic repercussions.
There is no cure for this disease but if it is identified and treated early, progression can be slowed or stopped.
The aim of this meeting is to give patients and caregivers the opportunity to share their lived experiences with CTX disease via live, moderated group discussions, and share perspectives on current and future treatments.
Following this meeting, a ”Voice of the patient” report will be provided to all persons affected by this disease and the scientific experts working on the CTX disease.