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Harmatz, Paul
508  Ergebnisse:
Personensuche X
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Design of a multi-center randomized active controlled phase..:

Orchard, Paul J. ; Wynn, Robert F. ; Kharbanda, Sandhya...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 107988 , 2024
Link: https://doi.org/10.1016/..
 
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2

Interim analysis of a phase 1/2 study of weekly intravenous..:

Muenzer, Joseph ; Burton, Barbara K. ; Harmatz, Paul...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 107972 , 2024
Link: https://doi.org/10.1016/..
 
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3

P141: Persistent growth-promoting effects of vosoritide in ..:

Savarirayan, Ravi ; Tofts, Louise ; Irving, Melita...
Genetics in Medicine Open.  2 (2024)  - p. 101038 , 2024
Link: https://doi.org/10.1016/..
 
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4

CAMPSIITE™ Phase I/II/III: An interim clinical study update..:

Pisani, Laura ; Ficicioglu, Can ; Harmatz, Paul...
Molecular Genetics and Metabolism.  141 (2024)  4 - p. 108245 , 2024
Link: https://doi.org/10.1016/..
 
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5

CAMPSIITE™ phase I/II/III: An interim clinical study update..:

Harmatz, Paul ; Ficicioglu, Can ; Giugliani, Roberto...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 107874 , 2024
Link: https://doi.org/10.1016/..
 
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6

A natural history study of late-infantile and juvenile GM1 ..:

Giugliani, Roberto ; Harmatz, Paul ; Héron, Bénédicte...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 107850 , 2024
Link: https://doi.org/10.1016/..
 
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7

P037: Design of a multi-center randomized phase 3 clinical ..:

Harmatz, Paul ; Wynn, Robert ; Gupta, Ashish...
Genetics in Medicine Open.  2 (2024)  - p. 100914 , 2024
Link: https://doi.org/10.1016/..
 
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8

P144: Persistence of growth-promoting effects in children w..:

Hoover-Fong, Julie ; Irving, Melita ; Bacino, Carlos...
Genetics in Medicine Open.  2 (2024)  - p. 101041 , 2024
Link: https://doi.org/10.1016/..
 
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9

Somatic outcomes in a phase 1/2 study of weekly intravenous..:

Burton, Barbara ; Muenzer, Joseph ; Harmatz, Paul...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 107782 , 2024
Link: https://doi.org/10.1016/..
 
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10

A natural history study of late-infantile and juvenile GM1 ..:

Giugliani, Roberto ; Harmatz, Paul ; Héron, Bénédicte...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 107849 , 2024
Link: https://doi.org/10.1016/..
 
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11

P139: Persistent growth-promoting effects of vosoritide in ..:

Savarirayan, Ravi ; Tofts, Louise ; Irving, Melita...
Genetics in Medicine Open.  2 (2024)  - p. 101036 , 2024
Link: https://doi.org/10.1016/..
 
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12

Design of a Multi-Center Randomized Active Controlled Phase..:

Gupta, Ashish O. ; Wynn, Robert F ; Kharbanda, Sandhya...
Transplantation and Cellular Therapy.  30 (2024)  2 - p. S236-S237 , 2024
Link: https://doi.org/10.1016/..
 
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13

A natural history study of late-infantile and juvenile GM1 ..:

Giugliani, Roberto ; Harmatz, Paul ; Héron, Bénédicte...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 107848 , 2024
Link: https://doi.org/10.1016/..
 
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14

Measurement of glycosaminoglycans in the amniotic fluid of ..:

Young, Sarah ; Lianoglou, Billie R. ; Herzeg, Akos...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 108090 , 2024
Link: https://doi.org/10.1016/..
 
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15

P131: Persistence of growth-promoting effects in infants an..:

Savarirayan, Ravi ; Wilcox, William ; Harmatz, Paul...
Genetics in Medicine Open.  2 (2024)  - p. 101028 , 2024
Link: https://doi.org/10.1016/..
 
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