A trial tests the technology of covid vaccines against a rare disease. Propionic acidemia is caused by mutations in the PCCA and PCBB genes.
These defects prevent the body from producing the enzymes necessary to break down food properly. In eight of the patients, the metabolic decompensations caused by the disease were reduced by 70%. Although side effects such as vomiting or diarrhea were observed, the safety of the therapy is not considered to be an issue, the authors say. But the authors recognize that the small number of patients treated makes it difficult to assess whether the results are significant.