First‐In‐Human Safety, Tolerability, and Pharmacokinetics of PPI‐1011, a Synthetic Plasmalogen Precursor
ABSTRACT PPI‐1011 is a synthetic plasmalogen precursor designed to augment plasmalogen levels in patients with Rhizomelic chondrodysplasia punctata (RCDP), an ultra‐rare genetic disorder caused by a plasmalogen deficiency that results in significant physical and mental delays. We report here a Phase...
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| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
Wiley
2025-03-01
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| Series: | Clinical and Translational Science |
| Subjects: | |
| Online Access: | https://doi.org/10.1111/cts.70195 |
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