Publications by authors named "John P Throup"
BACKGROUNDThe toxic accumulation of phenylalanine (Phe) in the brain underlies the neurological presentation of phenylketonuria (PKU). Solute carrier family 6 member 19 (SLC6A19) is the major transporter responsible for the (re)absorption of Phe in the kidney and intestine. Here, we describe the characterization of the first small molecule SLC6A19 inhibitor to enter clinical development for the treatment of PKU.
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- Urotensin II (U-II) is thought to play a role in lung function, and researchers tested U-T receptor antagonist GSK1440115 as a potential asthma treatment.
- A Phase I trial showed GSK1440115 was safe and well-tolerated in healthy adults, while a Phase Ib trial indicated asthmatic patients achieved necessary drug concentrations but did not show improvement in breathing tests.
- The study concluded that while the drug is safe, it failed to provide bronchodilation or protect against bronchospasm in asthmatics, suggesting limited effectiveness as an asthma treatment.