Evaluation of long-term safety and efficacy with RAVPAL-PEG (BMN 165) for control of blood phenylalanine levels in adults with phenylketonuria (PKU)

被引：0

作者：

Longo, Nicola ^{[1
]}

Thomas, Janet A. ^{[2
]}

Wasserstein, Melissa P. ^{[3
]}

Burton, Barbara K. ^{[4
]}

Vockley, Jerry ^{[5
]}

Grange, Dorothy K. ^{[6
]}

Hillman, Richard ^{[7
]}

Harding, Cary ^{[8
]}

Dimmock, David ^{[9
]}

Shur, Natasha ^{[10
]}

Adams, Darius ^{[11
]}

Rizzo, William B. ^{[12
]}

Whitley, Chester ^{[13
]}

Goodin, Kara M. ^{[14
]}

Decker, Celeste ^{[15
]}

Bolt, Kendra ^{[15
]}

Chen, Yinpu ^{[15
]}

Schweighardt, Becky ^{[15
]}

Zori, Roberto ^{[16
]}

机构：

[1] Univ Utah, Salt Lake City, UT USA

[2] Univ Colorado, Aurora, CO USA

[3] Mt Sinai Hosp, New York, NY 10029 USA

[4] Lurie Childrens Hosp, Chicago, IL USA

[5] Childrens Hosp Pittsburgh, Pittsburgh, PA 15213 USA

[6] St Louis Childrens Hosp, St Louis, MO 63178 USA

[7] Univ Missouri, Columbia, MO USA

[8] Oregon Hlth Sci, Portland, OR USA

[9] Med Coll Wisconsin, Milwaukee, WI 53226 USA

[10] Albany Med Coll, Albany, NY 12208 USA

[11] Albany Med Ctr, Albany, NY USA

[12] Nebraska Med Ctr, Omaha, NE USA

[13] Univ Minnesota, Minneapolis, MN USA

[14] Weisskopf Child Evaluat Ctr, Louisville, KY USA

[15] BioMarin Pharmaceut Inc, Novato, CA USA

[16] Univ Florida, Gainesville, FL USA

来源：

MOLECULAR GENETICS AND METABOLISM | 2015年 / 114卷 / 03期

关键词：

D O I：

暂无

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：365 / 366

页数：2

共 9 条

[1] Evaluation of an induction, titration, and maintenance dosing regimen in a phase 2 study of RAVPAL-PEG (BMN 165) for control of blood phenylalanine levels in adults with phenylketonuria (PKU)
Zori, Roberto
Thomas, Janet A.
Shur, Natasha
Rizzo, William B.
Decker, Celeste
Merilainen, Markus
Li, Mingjin
Schweighart, Becky
Longo, Nicola
MOLECULAR GENETICS AND METABOLISM, 2015, 114 (03) : 368 - 369
[2] Evaluation of multiple dosing regimens in phase 2 studies of rAvPAL-PEG (BMN 165) for control of blood phenylalanine levels in adults with phenylketonuria
Thomas, Janet A.
Longo, Nicola
Zori, Roberto
Burton, Barbara K.
Wasserstein, Melissa
Grange, Dorothy K.
Vockley, Jerry
Hillman, Richard
Harding, Cary
Shur, Natasha
Adams, Darius
Rice, Gregory M.
Rizzo, William B.
Whitley, Chester
Goodin, Kara M.
McBride, Kim L.
Decker, Celeste
Merilainen, Markus
Li, Mingjin
Schweighardt, Becky
Dimmock, David P.
MOLECULAR GENETICS AND METABOLISM, 2015, 114 (03) : 364 - 365
[3] Executive Functions and Long-Term Metabolic Control in Adults with Phenylketonuria (PKU)
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Thiele, Alena G.
Rohde, Carmen
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Kiess, Wieland
Beblo, Skadi
METABOLITES, 2025, 15 (03)
[4] Frontal lobe-dependent functions in treated phenylketonuria: Blood phenylalanine concentrations and long-term deficits in adolescents young adults
Feldmann, R
Denecke, J
Grenzebach, M
Weglage, J
JOURNAL OF INHERITED METABOLIC DISEASE, 2005, 28 (04) : 445 - 455
[5] Long-Term Exposures to Urban Noise and Blood Pressure Levels and Control Among Older Adults
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Evans, Denis A.
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[6] Long-term safety and efficacy of pegvaliase for the treatment of phenylketonuria in adults: combined phase 2 outcomes through PAL-003 extension study
Nicola Longo
Roberto Zori
Melissa P. Wasserstein
Jerry Vockley
Barbara K. Burton
Celeste Decker
Mingjin Li
Kelly Lau
Joy Jiang
Kevin Larimore
Janet A. Thomas
Orphanet Journal of Rare Diseases, 13
[7] Long-term safety and efficacy of pegvaliase for the treatment of phenylketonuria in adults: combined phase 2 outcomes through PAL-003 extension study
Longo, Nicola
Zori, Roberto
Wasserstein, Melissa P.
Vockley, Jerry
Burton, Barbara K.
Decker, Celeste
Li, Mingjin
Lau, Kelly
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Larimore, Kevin
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[8] CLINICAL-EVALUATION OF MEASURING GLYCOSYLATED HEMOGLOBIN LEVELS FOR ASSESSING THE LONG-TERM BLOOD-GLUCOSE CONTROL IN DIABETICS
INADA, M
OISHI, M
NISHIKAWA, M
KURATA, S
IMURA, H
ENDOCRINOLOGIA JAPONICA, 1980, 27 (04): : 411 - 415
[9] Efficacy and Safety of Long-Term Intravenous Tranexamic Acid Administration for Blood Management in Revision Surgery for Femoral Shaft Nonunion: A Retrospective Case-Control Study
Wang, Zhimeng
Lu, Yao
Huang, Qiang
Xue, Hanzhong
Ran, Cheng
Wang, Qian
Ma, Teng
Zhang, Kun
Li, Zhong
Sun, Liang
CLINICAL AND APPLIED THROMBOSIS-HEMOSTASIS, 2021, 27

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