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收藏分享 收藏 Safety and efficacy of migalastat in adolescent patients with Fabry disease: Results from ASPIRE , a phase 3b, open-label, single-arm, 12-month clinical trial, and its open-label extension
Ramaswami, Uma; Font-Montgomery, Esperanza; Goker-Alpan, Ozlem; Ortiz, Damara; Sanchez-Valle, Amarilis; Whitley, Chester B.; Wilcox, William R.; Jiang, Hai; Lawson, Lee Ann; Vosk, Jennie; Yang, Haichen; Hopkin, Robert J. 分享 收藏 Clinical assessment of disease severity in patients with Fabry disease treated with pegunigalsidase alfa: An integrated analysisHughes, Derralynn; Linhart, Ales; Wallace, Eric L.; Wilcox, William R.; Whitley, Chester B.; Vujkovac, Bojan; Koulinska, Irene; Piotti, Giovanni; Chertkoff, Raul; Alon, Sari; Sakov, Anat; Pisani, Antonio; Bernat, John A. 分享 收藏 Long-term safety and efficacy of migalastat in adolescent patients with Fabry disease: Results from the ASPIRE study and open-label extensionRamaswami, Uma; Font-Montgomery, Esperanza E.; Goker-Alpan, Ozlem; Ortiz, Damara; Sanchez-Valle, Amarilis; Whitley, Chester B.; Wilcox, William R.; Jiang, Hai; Lawson, Lee Ann; Vosk, Jennie; Yang, Haichen; Hopkin, Robert J. 分享 收藏 Quality of life of migalastat-treated adolescents with Fabry disease: Results from the ASPIRE study and open-label extensionHopkin, Robert J.; Sanchez-Valle, Amarilis; Font-Montgomery, Esperanza E.; Goker-Alpan, Ozlem; Ortiz, Damara; Whitley, chester B.; Wilcox, William R.; Jiang, Hai; Lawson, Lee Ann; Vosk, Jennie; Yang, Haichen; Ramaswami, Uma 分享 收藏 分享 收藏 Extending the interval between pegunigalsidase alfa infusions in patients with Fabry disease: Five-year interim results from the ongoing BRIGHT51 studyBernat, John A.; Holida, Myrl; Waldek, Stephen; Wilcox, William R.; Longo, Nicola; Goker-Alpan, Ozlem; Wallace, Eric; Deegan, Patrick; Tondel, Camilla; Eyskens, Francois; Feldt-Rasmussen, Ulla; Hughes, Derralynn; Pisani, Antonio; Mehta, Ankit; Nedd, Khan; Warnock, David G.; Piotti, Giovanni; Wang, Meng; Almon-Brill, Einat; Alon, Sari; Chertkoff, Raul; Linhart, Ales 分享 收藏 分享 收藏 Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of lifeSavarirayan, Ravi; Irving, Melita; Wilcox, William R.; Bacino, Carlos A.; Hoover-Fong, Julie E.; Harmatz, Paul; Polgreen, Lynda E.; Mohnike, Klaus; Prada, Carlos E.; Kubota, Takuo; Arundel, Paul; Leiva-Gea, Antonio; Rowell, Richard; Low, Andrea; Sabir, Ian; Huntsman-Labed, Alice; Day, Jonathan 分享 收藏 Isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)Hopkin, Robert J.; Ganesh, Jaya; Bernat, John; Goker-Alpan, Ozlem; Nicholls, Kathleen; Pahl, Madeleine V.; Deegan, Patrick B.; Whitley, Chester B.; Hughes, Derralynn; Cao, Liching; Chen, Michael; Shiue, Lisa; Rojkjaer, Lisa; Wilcox, William R. 分享 收藏 Characteristics and management of US pediatric patients with Gaucher disease from the Gaucher Outcome SurveyGoker-Alpan, Ozlem; Burrow, Andrew; Enns, Gregory M.; Ficicioglu, Can; Kishnani, Priya S.; Ortiz, Damara; Prada, Carlos E.; Vats, Divya; Weinreb, Neal J.; Robinson, Ian; Pathak, Ravi R.; Wright, Ekaterina; Botha, Jaco; Wilcox, William R. 分享 收藏 Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trialSavarirayan, Ravi; Wilcox, William R.; Harmatz, Paul; Phillips, John; Polgreen, Lynda E.; Tofts, Louise; Ozono, Keiichi; Arundel, Paul; Irving, Melita; Bacino, Carlos A.; Basel, Donald; Bober, Michael B.; Charrow, Joel; Mochizuki, Hiroshi; Kotani, Yumiko; Saal, Howard M.; Army, Clare; Jeha, George; Qi, Yulan; Han, Lynn; Fisheleva, Elena; Huntsman-Labed, Alice; Day, Jonathan 分享 收藏 Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE studyWallace, Eric L.; Goker-Alpan, Ozlem; Wilcox, William R.; Holida, Myrl; Bernat, John; Longo, Nicola; Linhart, Ales; Hughes, Derralynn A.; Hopkin, Robert J.; Tondel, Camilla; Langeveld, Mirjam; Giraldo, Pilar; Pisani, Antonio; Germain, Dominique Paul; Mehta, Ankit; Deegan, Patrick B.; Molnar, Maria Judit; Ortiz, Damara; Jovanovic, Ana; Muriello, Michael; Barshop, Bruce A.; Kimonis, Virginia; Vujkovac, Bojan; Nowak, Albina; Geberhiwot, Tarekegn; Kantola, Ilkka; Knoll, Jasmine; Waldek, Stephen; Nedd, Khan; Karaa, Amel; Brill-Almon, Einat; Alon, Sari; Chertkoff, Raul; Rocco, Rossana; Sakov, Anat; Warnock, David G. 分享 收藏 Vosoritide Therapy in Children with Achondroplasia: Early Experience and Practical Considerations for Clinical PracticeSemler, Oliver; Cormier-Daire, Valerie; Lausch, Ekkehart; Bober, Michael B.; Carroll, Ricki; Sousa, Sergio B.; Deyle, David; Faden, Maha; Hartmann, Gabriele; Huser, Aaron J.; Legare, Janet M.; Mohnike, Klaus; Rohrer, Tilman R.; Rutsch, Frank; Smith, Pamela; Travessa, Andre M.; Verardo, Angela; White, Klane K.; Wilcox, William R.; Hoover-Fong, Julie 分享 收藏 Global reach of over 20 years of experience in the patient-centered Fabry Registry: Advancement of Fabry disease expertise and dissemination of real-world evidence to the Fabry communityWanner, Christoph; Ortiz, Alberto; Wilcox, William R.; Hopkin, Robert J.; Johnson, Jack; Ponce, Elvira; Ebels, Johan T.; Batista, Julie L.; Maski, Manish; Politei, Juan M.; Martins, Ana Maria; Banikazemi, Maryam; Linhart, Ales; Mauer, Michael; Oliveira, Joao P.; Weidemann, Frank; Germain, Dominique P. 分享 收藏 分享 收藏 Clinical outcomes among young patients with Fabry disease who initiated agalsidase beta treatment before 30 years of age: An analysis from the Fabry RegistryHopkin, Robert J.; Cabrera, Gustavo H.; Jefferies, John L.; Yang, Meng; Ponce, Elvira; Brand, Eva; Feldt-Rasmussen, Ulla; Germain, Dominique P.; Guffon, Nathalie; Jovanovic, Ana; Kantola, Ilkka; Karaa, Amel; Martins, Ana M.; Tondel, Camilla; Wilcox, William R.; Yoo, Han-Wook; Burlina, Alessandro P.; Mauer, Michael 分享 收藏 STAAR, a phase I/II study of isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Dose escalation phase resultsHopkin, Robert J.; Ganesh, Jaya; Deegan, Patrick; Goker-Alpan, Ozlem; Bernat, John; Wilcox, William R.; Pahl, Madeleine V.; Whitley, Chester B.; Hughes, Derralynn; Nicholls, Kathleen; Cao, Liching; Chen, Michael; Shiue, Lisa H.; Bowden, Emma; Jaggumantri, Sravan; Passalacqua, Cristobal; Souberbielle, Bernard; Cockroft, Bettina M. 分享 收藏 Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension studyDeegan, Patrick B.; Goker-Alpan, Ozlem; Geberhiwot, Tarekegn; Hopkin, Robert J.; Lukina, Elena; Tylki-Szymanska, Anna; Zaher, Atef; Sensinger, Charlotte; Gaemers, Sebastiaan J. M.; Modur, Vijay; Thurberg, Beth L.; Sharma, Jyoti; Najafian, Behzad; Mauer, Michael; DasMahapatra, Pronabesh; Wilcox, William R.; Germain, Dominique P. 分享 收藏 
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