Fachrepositorium Lebenswissenschaften

2 Treffer

Kurzansicht	E-Jahr	Zugriff	Typ	Operations
Challenges in multinational rare disease clinical studies during COVID-19: regulatory assessment of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease 2025 . Schoser, Benedikt \| Attarian, Shahram \| Graham, Ryan \| Holdbrook, Fred \| Goldman, Mitchell \| Díaz-Manera, Jordi \| The ATB200-03 study group	2025		http://purl.org/ontology/bibo/Article
Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02) 2023 . Byrne, Barry J. \| Schoser, Benedikt \| Kishnani, Priya S. \| Bratkovic, Drago \| Clemens, Paula R. \| Goker-Alpan, Ozlem \| Ming, Xue \| Roberts, Mark \| Vorgerd, Matthias \| Sivakumar, Kumaraswamy \| van der Ploeg, Ans T. \| Goldman, Mitchell \| Wright, Jacquelyn \| Holdbrook, Fred \| Jain, Vipul \| Benjamin, Elfrida R. \| Johnson, Franklin \| Das, Sheela Sitaraman \| Wasfi, Yasmine \| Mozaffar, Tahseen	2023		http://purl.org/ontology/bibo/Article

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