Clinical Trials News & Updates

PMD Clinical Study

Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356…

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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 involved. 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 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.

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Please refer to the links below for the most up to date information on which clinical trials are recruiting new patients or in progress. Reach out to the facilitator of the trial directly, in order to determine eligibility for the trial. 

Clinicaltrials.gov
Clinical Trails Register (Europe)