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Long-term safety outcomes of eliglustat in patients with Gaucher disease: Prospective, multi-center, observational, post authorization safety sub-registry study Giraldo, Pilar; Batsu, Isabela; Accomando, Beverly; Gaemers, Sebastiaan J. M.; Cil, Sefika Uslu; Gouyette, Najat; Scarpa, Maurizio Share Save
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Learnings about Aβ from human brain recommend the use of a live-neuron bioassay for the discovery of next generation Alzheimer's disease immunotherapeutics Wang, Zemin; Jin, Ming; Hong, Wei; Liu, Wen; Reczek, David; Lagomarsino, Valentina N.; Hu, Yuan; Weeden, Tim; Frosch, Matthew P.; Young-Pearse, Tracy L.; Pradier, Laurent; Selkoe, Dennis; Walsh, Dominic M. Share Save
Glucosylsphingosine (Lyso-Gb1) as a reliable biomarker in Gaucher disease: a narrative review Giuffrida, Gaetano; Markovic, Uros; Condorelli, Annalisa; Calafiore, Valeria; Nicolosi, Daniela; Calagna, Marianna; Grasso, Stephanie; Ragusa, Marco Tindaro Valentino; Gentile, Jennifer; Napolitano, Mariasanta Share Save
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Progression-free survival and quality of life in metastatic breast cancer: The patient perspective Mertz, Shirley; Benjamin, Christine; Girvalaki, Charis; Cardone, Antonella; Gono, Paulina; May, Suepattra G.; Comerford, Erin; Than, Kyi-Sin; Birch, Kelly; Roach, Meaghan; Myers, Sky; Sasane, Medha; Lavi, Liat; Cameron, Anna; Cardoso, Fatima Share Save
The Family Impact of Atopic Dermatitis in the Pediatric Population: Results from an International Cross-sectional Study Barbarot, Sebastien; Silverberg, Jonathan, I; Gadkari, Abhijit; Simpson, Eric L.; Weidinger, Stephan; Mina-Osorio, Paola; Rossi, Ana B.; Brignoli, Lysel; Mnif, Tarek; Guillemin, Isabelle; Fenton, Miriam C.; Pellan, Marine; Mahajan, Puneet; Delevry, Dimittri; Bansal, Ashish; Eckert, Laurent Share Save
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Safety, pharmacokinetics and target engagement of novel RIPK1 inhibitor SAR443060 (DNL747) for neurodegenerative disorders: Randomized, placebo-controlled, double-blind phase I/Ib studies in healthy subjects and patients Vissers, Maurits F. J. M.; Heuberger, Jules A. A. C.; Groeneveld, Geert Jan; Nijhuis, Jerome Oude; De Deyn, Peter Paul; Hadi, Salah; Harris, Jeffrey; Tsai, Richard M.; Cruz-Herranz, Andres; Huang, Fen; Tong, Vincent; Erickson, Rebecca; Zhu, Yuda; Scearce-Levie, Kimberly; Hsiao-Nakamoto, Jennifer; Tang, Xinyan; Chang, Megan; Fox, Brian M.; Pomponio, Robert J.; Alonso-Alonso, Miguel; Zilberstein, Moshe; Atassi, Nazem; Troyer, Matthew D.; Ho, Carole Share Save
Dupilumab Treatment Reduces Hospitalizations in Adults With Moderate-to-Severe Atopic Dermatitis Silverberg, Jonathan, I; Rubini, Norma P. M.; Pires, Mario C.; Rossi, Ana B.; Zhang, Annie; Chen, Zhen; Levit, Noah A.; Chao, Jingdong; Shumel, Brad; Bego-Le Bagousse, Gaelle Share Save
Adjuvant study of amcenestrant (SAR439859) versus tamoxifen for patients with hormone receptor-positive (HR plus ) early breast cancer (EBC), who have discontinued adjuvant aromatase inhibitor therapy due to treatment-related toxicity (AMEERA-6) Rossi, G.; Brain, E.; Dueck, A.; De Swert, H.; Marreaud, S. I.; Partridge, A.; Herold, C.; Vachon, H.; Spanic, T.; Arahmani, A.; Verbiest, T.; Wang, L.; Goulioti, T.; Malanda, B.; Carey, L. A.; Anneheim, S.; Paux, G.; Poncet, C.; Metzger, O.; Cameron, D. Share Save
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Global intercompany assessment of ICIEF platform comparability for the characterization of therapeutic proteins Madren, Seth; McElroy, Will; Schultz-Kuszak, Kristin; Boumajny, Boris; Shu, Yao; Sautter, Sabine; Zhao, Helen C.; Schadock-Hewitt, Abby; Chumsae, Chris; Ball, Nancy; Zhang, Xiaoying; Rish, Kimberly; Zhang, Shukui; Wurm, Christine; Cai, Sumin; Bauer, Scott P.; Stella, Cinzia; Zheng, Laura; Roper, Brian; Michels, David A.; Wu, Gang; Kocjan, Bostjan; Birk, Matej; Erdmann, Simon Erik; He, Xiaoping; Whittaker, Brad; Song, Yvonne; Barrett, Hannah; Strozyk, Kevin; Jing, Ye; Huang, Long; Mhatre, Vishal; McLean, Paul; Yu, Tiantian; Yang, Huijuan; Mattila, Minna Share Save
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Safety, Pharmacokinetics, and Pharmacodynamics of Oral Venglustat in Patients with Parkinson's Disease and a GBA Mutation: Results from Part 1 of the Randomized, Double-Blinded, Placebo-Controlled MOVES-PD Trial Peterschmitt, M. Judith; Saiki, Hidemoto; Hatano, Taku; Gasser, Thomas; Isaacson, Stuart H.; Gaemers, Sebastiaan J. M.; Minini, Pascal; Saubadu, Stephane; Sharma, Jyoti; Walbillic, Samantha; Alcalay, Roy N.; Cutter, Gary; Hattori, Nobutaka; Hoeglinger, Gunter U.; Marek, Kenneth; Schapira, Anthony H., V; Scherzer, Clemens R.; Simuni, Tanya; Giladi, Nir; Sardi, Sergio Pablo; Fischer, Tanya Z. Share Save
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