検索結果 - Robin Lachmann

O33: Impact of p.Arg610del neuroprotective genotype on disease burden and treatment response in adults with acid-sphingomyelinase deficiency: ASCEND olipudase alfa trial 著者: Melissa Wasserstein, Monica Kumar, Nicole Armstrong, Sefika Uslu Cil, Arya Firoozan, Lisa Underhill, Robin Lachmann

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P349: Plasma lyso-sphingomyelin, biomarker for acid sphingomyelinase deficiency: Correlations with baseline disease and response to olipudase alfa treatment in clinical trials 著者: Melissa Wasserstein, Roberto Giugliani, Simon Jones, Robin Lachmann, Maurizio Scarpa, Monica Kumar, Nicole Armstrong, Mario Aguiar

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Enzyme replacement therapy for the treatment of late onset Pompe disease: A systematic review and network meta-analysis 著者: Mark Corbett, Chinyereugo Umemneku-Chikere, Sarah Nevitt, Nyanar Jasmine Deng, Matthew Walton, Helen Fulbright, Chong Yew Tan, Robin Lachmann, Rachel Churchill, Robert Hodgson

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Enzyme replacement therapy compared with best supportive care for the treatment of Pompe Disease: a systematic review and network meta-analysis 著者: Mark Corbett, Chinyereugo Umemneku-Chikere, Sarah Nevitt, Nyanar Jasmine Deng, Matthew Walton, Helen Fulbright, Chong Yew Tan, Robin Lachmann, Rachel Churchill, Robert Hodgson

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P206: Continued clinical improvements in adults with acid sphingomyelinase deficiency after 3-5 Years of olipudase alfa treatment: Final ASCEND trial results 著者: Melissa Wasserstein, Renata Gallagher, Antonio Barbato, Roberto Giugliani, Norberto Guelbert, Julia Hennermann, Takayuki Ikezoe, Robin Lachmann, Olivier Lidove, Paulina Mabe, Eugen Mengel, Maurizio Scarpa, Ebubekir Senates, Michel Tchan, Jesus Villarrubia, Beth Thurberg, Nicole Armstrong, Shruti Srivastava, Urszula Wisniewska, Monica Kumar

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Continued improvement in disease manifestations of acid sphingomyelinase deficiency for adults with up to 2 years of olipudase alfa treatment: open-label extension of the ASCEND tr... 著者: Melissa P. Wasserstein, Robin Lachmann, Carla Hollak, Antonio Barbato, Renata C. Gallagher, Roberto Giugliani, Norberto Bernardo Guelbert, Julia B. Hennermann, Takayuki Ikezoe, Olivier Lidove, Paulina Mabe, Eugen Mengel, Maurizio Scarpa, Ebubekir Senates, Michel Tchan, Jesus Villarrubia, Beth L. Thurberg, Abhimanyu Yarramaneni, Nicole M. Armstrong, Yong Kim, Monica Kumar

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