The primary purpose of this study is to evaluate the safety and tolerability of ION356.
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
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Ionis Investigative Site, Atlanta, Georgia, United States, 30342
Ionis Investigative Site, Philadelphia, Pennsylvania, United States, 19104
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
For general information about clinical research, read Learn About Studies.
2 Years to 17 Years
MALE
No
Ionis Pharmaceuticals, Inc.,
2028-06