POM-001 phase 1/2 study of BMN 701, GILT-tagged recombinant human (rh) GAA in late-onset Pompe disease: Initial experience in 22 patients

被引：1

作者：

Byrne, Barry ^{[1
]}

Barohn, Richard ^{[2
]}

Barshop, Bruce ^{[3
]}

Bratkovic, Drago ^{[4
]}

Desnuelle, Claude ^{[5
]}

Geberhiwot, Tarekegn ^{[6
,7
]}

Hughes, Derralynn ^{[8
]}

Laforet, Pascal ^{[9
]}

Mengel, Eugen ^{[10
]}

Roberts, Mark ^{[11
]}

Lang, William ^{[12
]}

LeBowitz, Jonathan ^{[12
]}

机构：

[1] Univ Florida, Gainesville, FL USA

[2] Univ Kansas, Med Ctr, Kansas City, MO USA

[3] Univ Calif San Diego, Med Ctr, San Diego, CA 92103 USA

[4] Womens & Childrens Hosp, Adelaide, SA, Australia

[5] Ctr Hosp Univ Hop Archet 1, Nice, France

[6] Univ Birmingham, Birmingham, W Midlands, England

[7] Queen Elizabeth Hosp Birmingham, Birmingham, W Midlands, England

[8] Royal Free London Hosp NHS Trust, London, England

[9] Hop La Pitie Salpetriere, Paris, France

[10] Univ Mainz Klinikum, Mainz, Germany

[11] Salford Royal Hosp NHS Trust, Manchester, Lancs, England

[12] BioMarin Pharmaceut Inc, Novato, CA 94949 USA

来源：

MOLECULAR GENETICS AND METABOLISM | 2013年 / 108卷 / 02期

关键词：

D O I：

10.1016/j.ymgme.2012.11.048

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：S28 / S28

页数：1

共 5 条

[1] POM-001 Phase 1/2 Study of BMN 701, GILT-tagged Recombinant Human (rh) GAA in Late-Onset Pompe Disease: Preliminary Report
Byrne, Barry
Barshop, Bruce
Barohn, Richard
Falk, Lindsay
Fox, Michael
Lang, William
Lossius, Michelle
McCarthy, Sean
Vora, Jayesh
LeBowitz, Jonathon
[J]. MOLECULAR GENETICS AND METABOLISM, 2012, 105 (02) : S24 - S24
[2] Preliminary clinical efficacy and safety of BMN 701, GILT-tagged recombinant human acid alpha glucosidase (rhGAA), in late-onset Pompe disease: results of an extension study
Byrne, Barry
Barohn, Richard
Barshop, Bruce
Bratkovic, Drago
Desnuelle, Claude
Henderson, Robert
Hiwot, Tarekegn
Hughes, Derralynn
Laforet, Pascal
Mengel, Eugen
Roberts, Mark
Lang, William
LeBowitz, Jonathon
[J]. MOLECULAR GENETICS AND METABOLISM, 2014, 111 (02) : S29 - S29
[3] Reveglucosidase alfa (BMN 701), a GILT-tagged recombinant human acid alpha glucosidase (rhGAA), evaluation in late-onset Pompe disease: Preliminary clinical efficacy and safety results of an extension study (72-week results)
Hiwot, T.
Barohn, R.
Bratkovic, D.
Byrne, B.
Desnuelle, C.
Hughes, D.
Laforet, P.
Mengel, E.
Roberts, M.
Yang, K.
Heusner, C.
Dummer, W.
[J]. NEUROMUSCULAR DISORDERS, 2015, 25 : S313 - S314
[4] Phase 1 Study of Gene Therapy in Late-Onset Pompe Disease: Initial 104-Week Experience
Smith, Edward C.
Hopkins, Sam
Case, Laura E.
Childers, Martin K.
Han, Sang-Oh
Spears, Tracy G.
Hornik, Christoph P.
Bali, Deeksha
Kishnani, Priya S.
Koeberl, Dwight D.
[J]. MOLECULAR THERAPY, 2022, 30 (04) : 563 - 564
[5] Administration of recombinant human acid alpha glucosidase (Rh-GAA) to patients with infantile Pompe disease results in a rapid decrease in left ventricular hypertrophy (LVH): Preliminary results from a phase 2 study
Levine, JC
Colan, S
Kishnani, P
Amalfitano, A
Tsai, CH
Herman, G
Waterson, J
Rogers, RC
Yong, F
Chen, YT
[J]. CIRCULATION, 2002, 106 (19) : 521 - 521

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