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E

Eugen Mengel

Baylor University Medical Center
H指数:14
被引数:1,118

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荣誉奖项

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Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial

Arimoclomol在尼曼-皮克病C型中的长期疗效与安全性：NPC-002 48个月开放标签扩展试验2/3期的最终结果

Molecular Genetics and Metabolism
IF3.5
2025-07-070
PRE
Eugen Mengel; Rosalia M. Da Riol; Mireia Del Toro; Federica Deodato; Matthias Gautschi; Stephanie Grunewald; Sabine Weller Grønborg; Paul Harmatz; Julia B. Hennermann; Bénédicte Héron; Esther M. Maier; Saikat Santra; Reena Sharma; Anna Tylki-Szymanska; Malene Cording; Louise Himmelstrup; Sven Guenther; Christine í Dali
Trappsol® CycloTM (hpβCd) for the long-term treatment of Niemann Pick type C1: Efficacy and safety data from 4 clinical studies and the ongoing expanded access program
MOLECULAR GENETICS AND METABOLISM
IF3.5
2025-02-010
PRE
Hastings, Caroline A.; Ezgu, Fatih S.; Giugliani, Roberto; Kiec-Wilk, Beata; Pawlinski, Lukasz; Mengel, Eugen; Martin-Hernandez, Elena; Ucar, Seema; Sharma, Reena; Smith, Nicholas J. C.; Chien, Yin-Hsiu; Walterfang, Mark; Alsayed, Moeen; Goker-Alpan, Ozlem; Mendonca, Leonardo O.; Raiman, Julian A.; Spiegel, Ronen; Barone, Rita Maria Elisa; Burlina, Alberto; Calandra, Cristian R.; Bayarri, Jordi Gascon; Peters, Heidi; Murray, Bryan J.; Brecht, Andreas F.; Mejia, Joseph
Trial of N-Acetyl-L-Leucine in Niemann-Pick Disease Type C
NEW ENGLAND JOURNAL OF MEDICINE
IF78.5
2024-02-018
PRE
Bremova-Ertl, Tatiana; Ramaswami, Uma; Brands, Marion; Foltan, Tomas; Gautschi, Matthias; Gissen, Paul; Gowing, Francesca; Hahn, Andreas; Jones, Simon; Kay, Richard; Kolnikova, Miriam; Arash-Kaps, Laila; Marquardt, Thorsten; Mengel, Eugen; Park, Julien H.; Reichmannova, Stella; Schneider, Susanne A.; Sivananthan, Siyamini; Walterfang, Mark; Wibawa, Pierre; Strupp, Michael; Martakis, Kyriakos
Results of a phase III, randomized, placebo-controlled crossover trial with N-acetyl-L-leucine for Niemann-Pick disease type C
MOLECULAR GENETICS AND METABOLISM
IF3.5
2024-02-010
PRE
Fields, Taylor; Bremova-Ertl, Tatiana; Ramaswami, Uma; Brands, Marion; Gautschi, Matthias; Gissen, Paul; Hahn, Andreas; Jones, Simon; Kolnikova, Miriam; Arash-Karps, Laila; Mengel, Eugen; Park, Julien; Reichmannova, Stella; Schneider, Susanne; Sivananthan, Siyamini; Walterfang, Mark; Wibawa, Pierre; Strupp, Michael; Martakis, Kyriakos
Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease
NEUROLOGY
IF8.5
2022-08-0224
OAAI
Dimachkie, Mazen M.; Barohn, Richard J.; Byrne, Barry; Goker-Alpan, Ozlem; Kishnani, Priya S.; Ladha, Shafeeq; Laforet, Pascal; Mengel, Karl Eugen; Pena, Loren D. M.; Sacconi, Sabrina; Straub, Volker; Trivedi, Jaya; Van Damme, Philip; van der Ploeg, Ans T.; Vissing, John; Young, Peter; Haack, Kristina An; Foster, Meredith; Gilbert, Jane M.; Miossec, Patrick; Vitse, Olivier; Zhou, Tianyue; Schoser, Benedikt
Clinical disease progression and biomarkers in Niemann-Pick disease type C: a prospective cohort study (vol 15, 328, 2020)
ORPHANET JOURNAL OF RARE DISEASES
IF3.5
2021-06-011
OAAI
Mengel, Eugen; Bembi, Bruno; del Toro, Mireia; Deodato, Federica; Gautschi, Matthias; Grunewald, Stephanie; Gronborg, Sabine; Heron, Benedicte; Maier, Esther M.; Roubertie, Agathe; Santra, Saikat; Tylki-Szymanska, Anna; Day, Simon; Symonds, Tara; Hudgens, Stacie; Patterson, Marc C.; Guldberg, Christina; Ingemann, Linda; Petersen, Nikolaj H. T.; Kirkegaard, Thomas; Dali, Christine i
NEO1/NEO-EXT studies: Safety and exploratory efficacy of repeat avalglucosidase alfa dosing after up to 6 years in participants with late-onset pompe disease (LOPD)
MOLECULAR GENETICS AND METABOLISM
IF3.5
2021-02-011
PREAI
Dimachkie, Mazen M.; Barohn, Richard J.; Byrne, Barry; Goker-Alpan, Ozlem; Kishnani, Priya S.; Ladha, Shafeeq; Laforet, Pascal; Mengel, Karl Eugen; Pena, Loren D. M.; Sacconi, Sabrina; Straub, Volker; Trivedi, Jaya; Van Damme, Philip; van der Ploeg, Ans; Vissing, John; Young, Peter; Haack, Kristina An; Ivanina, Inna; Lu, Xiaoyu; Schoser, Benedikt
Clinical disease progression and biomarkers in Niemann-Pick disease type C: a prospective cohort study
ORPHANET JOURNAL OF RARE DISEASES
IF3.5
2020-11-2315
OAAI
Mengel, Eugen; Bembi, Bruno; del Toro, Mireia; Deodato, Federica; Gautschi, Matthias; Grunewald, Stephanie; Gronborg, Sabine; Heron, Benedicte; Maier, Esther M.; Roubertie, Agathe; Santra, Saikat; Tylki-Szymanska, Anna; Day, Simon; Symonds, Tara; Hudgens, Stacie; Patterson, Marc C.; Guldberg, Christina; Ingemann, Linda; Petersen, Nikolaj H. T.; Kirkegaard, Thomas; i Dali, Christine
NEO1 and NEO-EXT studies: Long-term safety and exploratory efficacy of repeat avalglucosidase alfa dosing for 5.5 years in late-onset Pompe disease patients
MOLECULAR GENETICS AND METABOLISM
IF3.5
2020-02-015
PREAI
Dimachkie, Mazen M.; Barohn, Richard J.; Byrne, Barry; Goker-Alpan, Ozlem; Kishnani, Priya S.; Ladha, Shafeeq; Laforet, Pascal; Mengel, Karl Eugen; Pena, Loren D. M.; Sacconi, Sabrina; Straub, Volker; Trivedi, Jaya; Van Damme, Philip; van der Ploeg, Ans; Vissing, John; Young, Peter; Haack, Kristina An; Fleurinck, Carmen; Johnson, Judith; Liu, Kejian; Schoser, Benedikt

研究方向

暂未获取到该数据
R
Roberto Giugliani
H 指数:80论文数:1,417被引数:2.4万
S
Simon Jones
H 指数:59论文数:502被引数:1.0万
M
Matthias Gautschi
H 指数:28论文数:99被引数:4,166
B
Bruno Bembi
H 指数:53论文数:200被引数:8,351
N
Nathalie Guffon
H 指数:47论文数:262被引数:1.0万
P
Paul Harmatz
H 指数:65论文数:532被引数:1.4万
M
Marc C. Patterson
H 指数:62论文数:358被引数:1.3万
C
Christine í Dali
H 指数:20论文数:104被引数:1,091
R
Raphael Schiffmann
H 指数:90论文数:448被引数:2.4万
Ö
Özlem Göker-Alpan
H 指数:41论文数:262被引数:6,209