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Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study
DA Hughes, K Nicholls, SP Shankar, G Sunder-Plassmann, D Koeller, ...
Journal of medical genetics 54 (4), 288-296, 2017
4302017
Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 …
DP Germain, R Giugliani, DA Hughes, A Mehta, K Nicholls, L Barisoni, ...
Orphanet journal of rare diseases 7, 1-11, 2012
1662012
Seladelpar (MBX-8025), a selective PPAR-δ agonist, in patients with primary biliary cholangitis with an inadequate response to ursodeoxycholic acid: a double-blind, randomised …
D Jones, PF Boudes, MG Swain, CL Bowlus, MR Galambos, BR Bacon, ...
The lancet Gastroenterology & hepatology 2 (10), 716-726, 2017
1632017
A phase 2 study of migalastat hydrochloride in females with Fabry disease: selection of population, safety and pharmacodynamic effects
R Giugliani, S Waldek, DP Germain, K Nicholls, DG Bichet, JK Simosky, ...
Molecular Genetics and Metabolism 109 (1), 86-92, 2013
1272013
Drug compliance in therapeutic trials: a review
P Boudes
Controlled clinical trials 19 (3), 257-268, 1998
1121998
A pharmacogenetic approach to identify mutant forms of α‐galactosidase a that respond to a pharmacological chaperone for Fabry disease
X Wu, E Katz, MCD Valle, K Mascioli, JJ Flanagan, JP Castelli, ...
Human mutation 32 (8), 965-977, 2011
962011
A randomized, double‐blind, active‐and placebo‐controlled efficacy and safety study of arhalofenate for reducing flare in patients with gout
J Poiley, AS Steinberg, YJ Choi, CS Davis, RL Martin, CA McWherter, ...
Arthritis & Rheumatology 68 (8), 2027-2034, 2016
802016
Oral migalastat HCl leads to greater systemic exposure and tissue levels of active α-galactosidase A in Fabry patients when co-administered with infused agalsidase
DG Warnock, DG Bichet, M Holida, O Goker-Alpan, K Nicholls, M Thomas, ...
PLoS One 10 (8), e0134341, 2015
762015
The challenges of new drugs benefits and risks analysis: lessons from the ximelagatran FDA Cardiovascular Advisory Committee
PF Boudes
Contemporary clinical trials 27 (5), 432-440, 2006
622006
Migalastat HCl Reduces Globotriaosylsphingosine (Lyso-Gb3) in Fabry Transgenic Mice and in the Plasma of Fabry Patients
B Young-Gqamana, N Brignol, HH Chang, R Khanna, R Soska, M Fuller, ...
PLoS One 8 (3), e57631, 2013
552013
An open‐label study to determine the pharmacokinetics and safety of migalastat HCl in subjects with impaired renal function and healthy subjects with normal renal function
FK Johnson, PN Mudd Jr, T DiMino, J Vosk, S Sitaraman, P Boudes, ...
Clinical Pharmacology in Drug Development 4 (4), 256-261, 2015
542015
Duvoglustat HCl increases systemic and tissue exposure of active acid α-glucosidase in Pompe patients co-administered with alglucosidase α
P Kishnani, M Tarnopolsky, M Roberts, K Sivakumar, M Dasouki, ...
Molecular Therapy 25 (5), 1199-1208, 2017
522017
The pharmacodynamics, pharmacokinetics, and safety of arhalofenate in combination with febuxostat when treating hyperuricemia associated with gout
AS Steinberg, BD Vince, YJ Choi, RL Martin, CA McWherter, PF Boudes
The Journal of rheumatology 44 (3), 374-379, 2017
442017
A phase II, randomized, open-label, 52-week study of seladelpar in patients with primary biliary cholangitis
CL Bowlus, MR Galambos, RJ Aspinall, GM Hirschfield, DEJ Jones, ...
Journal of hepatology 77 (2), 353-364, 2022
422022
Pharmacokinetics and safety of migalastat HCl and effects on agalsidase activity in healthy volunteers
FK Johnson, PN Mudd Jr, A Bragat, M Adera, P Boudes
Clinical Pharmacology in Drug Development 2 (2), 120-132, 2013
422013
Gene therapy as a new treatment option for inherited monogenic diseases
PF Boudes
European journal of internal medicine 25 (1), 31-36, 2014
402014
TSE-424, a novel tissue selective estrogen, reduces biochemical indices of bone metabolism in a dose related fashion.
S Ronkin, L Clarke, P Boudes, G Constantine
JOURNAL OF BONE AND MINERAL RESEARCH 16, S413-S413, 2001
362001
Seladelpar efficacy and safety at 3 months in patients with primary biliary cholangitis: ENHANCE, a phase 3, randomized, placebo-controlled study
GM Hirschfield, ML Shiffman, A Gulamhusein, KV Kowdley, JM Vierling, ...
Hepatology 78 (2), 397-415, 2023
262023
Novel quantitative method to evaluate globotriaosylceramide inclusions in renal peritubular capillaries by virtual microscopy in patients with fabry disease
L Barisoni, JC Jennette, R Colvin, S Sitaraman, A Bragat, J Castelli, ...
Archives of pathology & laboratory medicine 136 (7), 816-824, 2012
262012
A randomized controlled trial of a protease inhibitor (saquinavir) in combination with zidovudine in previously untreated patients with advanced HIV infection.
S Vella, A Lazzarin, G Carosi, A Sinicco, O Armignacco, G Angarano, ...
Antiviral therapy 1 (3), 129-140, 1996
261996
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