Phase 1 safety and pharmacokinetics of the novel enzyme replacement therapy neoGAA in treatment-naïve and alglucosidase alfa-treated late-onset Pompe disease patients

2016 ◽  
Vol 117 (2) ◽  
pp. S117-S118
Author(s):  
Shafeeq Ladha ◽  
Pascal Laforet ◽  
Eugen Mengel ◽  
Alan Pestronk ◽  
Jean Pouget ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Late Onset ◽  
Phase 1 ◽  
The Novel ◽  
Enzyme Replacement ◽  
Alglucosidase Alfa ◽  
Treatment Naïve

Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease

2017 ◽  
Vol 122 (1-2) ◽  
pp. 80-85 ◽  
Author(s):  
Constantinos Papadopoulos ◽  
David Orlikowski ◽  
Hélène Prigent ◽  
Arnaud Lacour ◽  
Céline Tard ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Late Onset ◽  
Enzyme Replacement ◽  
Alglucosidase Alfa

Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease

2016 ◽  
Vol 26 ◽  
pp. S109-S110 ◽  
Author(s):  
C. Papadopoulos ◽  
D. Orlikowski ◽  
H. Prigent ◽  
B. Perniconi ◽  
N. Taouagh ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Late Onset ◽  
Enzyme Replacement ◽  
Alglucosidase Alfa
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