New insights into pathogenesis of acute porphyria attacks and proof-of-concept of augmenting hepatic PBGD as etiological treatment
Acute Intermittent Porphyria (AIP) is a rare disease caused by a hepatic deficiency of porphobilinogen deaminase (PBGD) and is characterized by acute neurovisceral attacks. Currently, available treatments for AIP are not optimal and do not cover the entire spectrum of symptoms and presentations of t...
| Main Authors: | , |
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| Format: | info:eu-repo/semantics/doctoralThesis |
| Language: | eng |
| Published: |
Universidad de Navarra
2022
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| Subjects: | |
| Online Access: | https://hdl.handle.net/10171/63816 |