A white female infant had first been noted to be jaundiced shortly after birth. She was the product of an uncomplicated pregnancy in a 21-year-old gravida 3, para 2. The birth weight was 3.45 kg.
Infantile Pompe disease resulting from a deficiency of lysosomal acid α-glucosidase (GAA) requires enzyme replacement therapy (ERT) with recombinant human GAA (rhGAA). Cross-reactive immunologic ...
Phase I dose escalation of the oral histone deacetylase inhibitor (HDACi) resminostat in combination with FOLFIRI in colorectal cancer (CRC) patients: The SHORE trial.