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收藏Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial
Savarirayan, 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
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收藏Further delineation of auriculocondylar syndrome based on 14 novel cases and reassessment of 25 published cases
Vegas, Nancy; Demir, Zeynep; Gordon, Christopher T.; Breton, Sylvain; Tavares, Vanessa L. Romanelli; Moisset, Hugo; Zechi-Ceide, Roseli; Kokitsu-Nakata, Nancy M.; Kido, Yasuhiro; Marlin, Sandrine; Halem, Souad Gherbi; Meerschaut, Ilse; Callewaert, Bert; Chung, Brian; Revencu, Nicole; Lehalle, Daphne; Petit, Florence; Propst, Evan J.; Papsin, Blake C.; Phillips, John H.; Jakobsen, Linda; Le Tanno, Pauline; Thevenon, Julien; McGaughran, Julie; Gerkes, Erica H.; Leoni, Chiara; Kroisel, Peter; Tan, Tiong Y.; Henderson, Alex; Terhal, Paulien; Basel-Salmon, Lina; Alkindy, Adila; White, Susan M.; Passos-Bueno, Maria R.; Pingault, Veronique; De Pontual, Loic; Amiel, Jeanne
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收藏Management of acute hepatic porphyria attacks in europe and united states: EXPLORE international, prospective, natural history study
Sardh, Eliane; Balwani, Manisha; Gouya, Laurent; Bissell, D. Montgomery; Rees, David; Stoelzel, Ulrich; Phillips, John; Kauppinen, Raili; Langendonk, Janneke; Desnick, Robert; Deybach, Jean Charles; Bonkovsky, Herbert; Parker, Charles; Naik, Hetanshi; Badminton, Mike; Stein, Penelope; Minder, Elisabeth; Windyga, Jerzy; Martasek, Pavel; Cappellini, Maria Domenica; Ventura, Paolo; Harper, Pauline; Sandberg, Sverre; Aarsand, Aasne; Alegre, Felix; Ivanova, Aneta; Talbi, Neila; Ko, John; Cahana-Amitay, Dalia; Chan, Amy; Quinn Dinh; Penz, Craig; Simon, Amy; Lin, Tim; Anderson, Karl
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收藏Acute hepatic porphyria disease manifestations and daily life impacts in EXPLORE international, prospective, natural history study
Gouya, Laurent; Balwani, Manisha; Bissell, D. Montgomery; Rees, David; Stoelzel, Ulrich; Phillips, John; Kauppinen, Raili; Langendonk, Janneke; Desnick, Robert; Deybach, Jean Charles; Bonkovsky, Herbert; Parker, Charles; Naik, Hetanshi; Badminton, Michael; Stein, Penelope; Minder, Elisabeth; Windyga, Jerzy; Martasek, Pavel; Cappellini, Maria Domenica; Ventura, Paolo; Sardh, Eliane; Harper, Pauline; Sandberg, Sverre; Aarsand, Aasne; Alegre, Felix; Ivanova, Aneta; Talbi, Neila; Ko, John; Cahana-Amitay, Dalia; Chan, Amy; Quinn Dinh; Penz, Craig; Simon, Amy; Lin, Tim; Anderson, Karl
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收藏Early Implantation of Ventricular Assisted Devices (VADs) in Failing Single Ventricle Physiology Patients Can Lead to Better Outcomes in Pediatric Heart Transplantation
Shih, R.; Phillips, J.; Tsifansky, M.; Machado, D.; Ebraheem, M.; Venkata, G.; Sullivan, K.; Gupta, D.; Reyes, K.; Pietra, B.; Fricker, F. J.; Bleiweis, M.
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收藏PORPHYRIA CUTANEA TARDA: PROFILE OF 189 PATIENTS FROM THE PORPHYRIAS CONSORTIUM IN THE UNITED STATES
Singal, Ashwani K.; Naik, Hetanshi; Overbey, Jessica R.; Balwani, Manisha; Liu, Lawrence; Wang, Bruce; Parker, Charles; Keel, Sioban; Erwin, Angelika; Levy, Cynthia; Bloomer, Joseph R.; Arora, Sumant; Bissell, Dwight M.; Bonkovsky, Herbert L.; Phillips, John; Desnick, Robert; Anderson, Karl E.
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