Finding the cure is the answer; working together is the first step.
Take the step to join a clinical trial for leukodystrophy therapies, if you are able! Improved therapies and a cure will only be found if researchers have patients involved. With rare diseases, it is even more key to have every patient able to be involved providing assistance to the research process. Sometimes patients or caregivers are concerned about joining clinical trials because of the fear of “being a guinea pig to science.” Patients involved in clinical trials are not “guinea pigs.” They are human beings, and the researchers who rely on the patients for their participation are deeply grateful. Patients who are involved in clinical trials are first in line to receive potentially life changing, and lifesaving, treatments. Results of clinical trials also impact the future of every single person who faces a leukodystrophy diagnosis. The ULF hopes you will consider being part of clinical trials, as more become available for the leukodystrophies.
Please refer to the link below for the most up to date information on what clinical trials are recruiting or in progress. You will need to reach out to the facilitator of the trial directly in order to determine eligibility for the trial.
Below are the clinical trials that the ULF is proud to promote on behalf of our partners. If you see a clinical trial below that you believe you are eligible for, please reach out to Keely Mata, Director of Operations, at email@example.com. She will get you in touch with our contact for that trial. Please note: the ULF can only assist with connecting you to the trials associated with our partners below. If there is a trial on ClinicalTrials.gov that you are interested in, you will need to reach out directly to the facilitators of the trial.
The Guanabenz Trial for Vanishing White Matter
For more information, click here.
The Natural History Of Metachromatic Leukodystrophy (HOME) Study, hosted by NORD’s IAMRARE™ Registry Program, represents an opportunity to address an area of unmet need, providing dynamic data collection and a novel framework for building regulatory-grade rare disease natural history studies incorporating patient-reported information. For more information, click here.