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Mengel Eugen
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1

Qualitative Study of the Patient Experience with Venglustat..:

Schiffmann, Raphael ; Mengel, Eugen ; Wallace, Mary...
Advances in Therapy.  41 (2024)  7 - p. 2907-2923 , 2024
Link: https://doi.org/10.1007/..
 
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2

NEO1/NEO-EXT studies: Long-term muscle quantitative magneti..:

Byrne, Barry ; Carlier, Pierre G. ; Vissing, John...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 107783 , 2024
Link: https://doi.org/10.1016/..
 
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3

Assessment of health state utilities associated with adult ..:

Matza, Louis S. ; Stewart, Katie D. ; Fournier, Marie...
The European Journal of Health Economics.  , 2024
Link: https://doi.org/10.1007/..
 
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4

A retrospective study of morbidity and mortality of chronic..:

Mengel, Eugen ; Muschol, Nicole ; Weinhold, Natalie...
Orphanet Journal of Rare Diseases.  19 (2024)  1 - p. , 2024
Link: https://doi.org/10.1186/..
 
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5

Improvements in liver and lipid outcomes continue in childr..:

Lachmann, Robin ; Ganesh, Jaya ; Giugliani, Roberto...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 107927 , 2024
Link: https://doi.org/10.1016/..
 
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6

Development of a suspicion index tool to aid diagnosis of A..:

Wiesinger, Anna-Maria ; Lauth, Wanda ; Giugliani, Roberto...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 108082 , 2024
Link: https://doi.org/10.1016/..
 
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7

Transport®NPC: open phase 3 global trial of intravenous hyd..:

Hastings, Caroline A. ; Goker-Alpan, Ozlem ; Pena, Loren...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 107875 , 2024
Link: https://doi.org/10.1016/..
 
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8

Natural history of acid sphingomyelinase deficiency among E..:

Mengel, Eugen ; Scarpa, Maurizio ; Guffon, Nathalie...
European Journal of Medical Genetics.  70 (2024)  - p. 104954 , 2024
Link: https://doi.org/10.1016/..
 
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9

Start, switch and stop (triple‐S) criteria for enzyme repla..:

Schoser, Benedikt ; van der Beek, Nadine A. M. E. ; Broomfield, Alexander...
European Journal of Neurology.  , 2024
Link: https://doi.org/10.1111/..
 
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10

Results of a phase III, randomized, placebo-controlled cros..:

Fields, Taylor ; Bremova-Ertl, Tatiana ; Ramaswami, Uma...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 107827 , 2024
Link: https://doi.org/10.1016/..
 
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11

Consensus clinical management guidelines for acid sphingomy..:

Geberhiwot, Tarekegn ; Wasserstein, Melissa ; Wanninayake, Subadra...
Orphanet Journal of Rare Diseases.  18 (2023)  1 - p. , 2023
Link: https://doi.org/10.1186/..
 
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12

Correction : Long-term safety and clinical outcomes of olip..:

Diaz, George A. ; Giugliani, Roberto ; Gufon, Nathalie...
Orphanet Journal of Rare Diseases.  18 (2023)  1 - p. , 2023
Link: https://doi.org/10.1186/..
 
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13

P1483: LONG-TERM IMPACT OF OLIPUDASE ALFA ENZYME REPLACEMEN..:

Villarrubia, Jesus ; Ganesh, Jaya ; Giugliani, Roberto...
HemaSphere.  7 (2023)  S3 - p. e18031f6 , 2023
Link: https://doi.org/10.1097/..
 
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14

Natural history of acid sphingomyelinase deficiency (ASMD) ..:

Mengel, Eugen ; Scarpa, Maurizio ; Guffon, Nathalie...
Molecular Genetics and Metabolism.  138 (2023)  2 - p. 107224 , 2023
Link: https://doi.org/10.1016/..
 
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15

Continued improvement in disease manifestations of acid sph..:

Wasserstein, Melissa P. ; Lachmann, Robin ; Hollak, Carla...
Orphanet Journal of Rare Diseases.  18 (2023)  1 - p. , 2023
Link: https://doi.org/10.1186/..
 
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