Paul A. Fitzpatrick
BioMarin Pharmaceutical
12 Papers
113 Citations
Paul A. Fitzpatrick is an academic researcher from BioMarin Pharmaceutical. The author has contributed to research in topics: Phenylalanine ammonia-lyase & Biology. The author has an hindex of 10, co-authored 12 publications.
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Papers
Structural and biochemical characterization of the therapeutic Anabaena variabilis phenylalanine ammonia lyase.
Lin Wang,Alejandra Gámez,Holly Archer,Enrique E. Abola,Christineh N. Sarkissian,Paul A. Fitzpatrick,Dan J Wendt,Zhang Yanhong,Michel C. Vellard,Joshua R. Bliesath,Sean M. Bell,Jeffrey F. Lemontt,Charles R. Scriver,Raymond C. Stevens +13 more
TL;DR: A proposed mechanism in which the amino group of the phenylalanine substrate is attacked directly by the 4-methylidene-imidazole-5-one prosthetic group is proposed, which proposes a helix-to-loop conformational switch in the helices flanking the inner active-site loop that regulates accessibility of the active site.
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Development of Pegylated Forms of Recombinant Rhodosporidium toruloides Phenylalanine Ammonia-Lyase for the Treatment of Classical Phenylketonuria
Alejandra Gámez,Christineh N. Sarkissian,Lin Wang,Woomi Kim,Mary Straub,Marianne G. Patch,Lin Chen,Steve Striepeke,Paul A. Fitzpatrick,Jeffrey F. Lemontt,Charles A. O'Neill,Charles R. Scriver,Raymond C. Stevens +12 more
TL;DR: From a series of formulations of linear and branched polyethylene glycols chemically conjugated to PAL, a parenteral therapeutic agent for PKU treatment is created, suggesting potential as a novel PKU therapeutic.
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Erythrocyte-mediated delivery of phenylalanine ammonia lyase for the treatment of phenylketonuria in BTBR-Pahenu2 mice
Luigia Rossi,Francesca Pierigè,Claudia Carducci,Claudia Gabucci,Tiziana Pascucci,Barbara Canonico,Sean M. Bell,Paul A. Fitzpatrick,Vincenzo Leuzzi,Mauro Magnani +9 more
TL;DR: Investigating if erythrocytes loaded with PAL may act as a safe delivery system able to overcome bioavailability issues and to provide, in vivo, a therapeutically relevant concentration of enzyme showed that the therapeutic effect was persistent and not affected by the generation of antibodies induced by the recombinant enzyme.
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Structural basis for the inhibition of poly(ADP-ribose) polymerases 1 and 2 by BMN 673, a potent inhibitor derived from dihydropyridophthalazinone
TL;DR: BMN 673, a novel PARP1/2 inhibitor in clinical development with substantial tumor cytotoxicity, forms extensive hydrogen-bonding and π-stacking in the nicotinamide pocket, with its unique disubstituted scaffold extending towards the less conserved edges of the pocket.
Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice.
Mika Aoyagi-Scharber,Danielle Crippen-Harmon,Roger Lawrence,Jon Vincelette,Gouri Yogalingam,Heather Prill,Bryan K. Yip,Brian Baridon,Catherine Vitelli,Amanda Lee,Olivia Gorostiza,Evan G. Adintori,Wesley C. Minto,Jeremy L. Van Vleet,Bridget Yates,Sara Rigney,Terri Christianson,Pascale M.N. Tiger,Melanie J. Lo,John Holtzinger,Paul A. Fitzpatrick,Jonathan H. LeBowitz,Sherry Bullens,Brett E. Crawford,Stuart Bunting +24 more
TL;DR: The substantial brain uptake of NAGLU attainable by this highest ICV dosage was required for nearly complete attenuation of disease-driven storage accumulations and neuropathology throughout the Naglu−/− mouse brain.
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