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Showing items 1-6 of 6  (1 Page(s) Totally)
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Institution Date Title Author
臺大學術典藏 2022-03-28T02:45:55Z Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial Diaz-Manera J.; Kishnani P.S.; Kushlaf H.; Ladha S.; Mozaffar T.; Straub V.; Toscano A.; van der Ploeg A.T.; Berger K.I.; Clemens P.R.; YIN-HSIU CHIEN; Day J.W.; Illarioshkin S.; Roberts M.; Attarian S.; Borges J.L.; Bouhour F.; Choi Y.C.; Erdem-Ozdamar S.; Goker-Alpan O.; Kostera-Pruszczyk A.; Haack K.A.; Hug C.; Huynh-Ba O.; Johnson J.; Thibault N.; Zhou T.; Dimachkie M.M.; Schoser B.; Behin A.; Boentert M.; Carvalho G.; Chahin N.; Charrow J.; Deegan P.; Durmus Tekce H.; Duval F.; Genge A.; Gutmann L.; Henderson R.D.; Hennermann J.B.; Hiwot T.; Hughes D.; Karaa A.; Karam C.; Kautzky-Willer A.; Komaki H.; Laforet P.; Longo N.; Malinova V.; Mar? R.; Maxit C.; Mengel E.; Moggio M.G.; Moln?r M.J.; Mongini T.E.; Nadaj-Pakleza A.; Nascimento Osorio A.; Noury J.-B.; Oliveira A.S.B.; Parman Y.; Pena L.; Remiche G.; Sciacco M.; Shieh P.B.; Smith C.; Stulnig T.; Taithe F.; Tard C.; Tarnopolsky M.; Vorgerd M.; Whitley C.; Young P.; Alonso-P?rez J.; Altemus P.; Aub?-Nathier A.-C.; Avelar J.B.; Bailey C.; Bekircan-Kurt C.E.; Billy J.; Boschi S.; Brown K.E.; Carrera Garcia L.; Chase L.; Cirne H.; Danjoux L.; Davion J.-B.; DeArmey S.; Fedotova E.; Gandolfo E.; Grosz Z.; Guellec D.; Guettsches A.-K.; Guglieri M.; Hatcher E.; Helms S.; Hufgard-Leitner M.; Klyushnikov S.A.; Langton J.; Linkov? L.; Mavroudakis N.; Mazurov? S.; Mori M.; M?ller-Miny L.; Musumeci O.; Nance C.S.; Natera-de Benito D.; Neel R.; Niizawa G.A.; Noll L.; Ortega E.; Pasnoor M.; Pautot V.; Potulska-Chromik A.; Pugliese A.; Questienne C.; Ramos Lopes M.; Reyes-Leiva D.; Riedl M.; Rugiero M.F.; Salort-Campana E.; Sgobbi Souza P.V.; Sole G.; Solera L.; Souto Lopes S.; Specht S.; Statland J.; Swenson A.; Tan C.Y.; Tizon S.; van der Beek N.A.M.E.; van Kooten H.A.; Wencel M.; Wenninger S.; Zagnoli F.; COMET Investigator Group
臺大學術典藏 2022-02-18T06:49:23Z Throwing a spotlight on under-recognized manifestations of Gaucher disease: Pulmonary involvement, lymphadenopathy and Gaucheroma Ramaswami U.; Mengel E.; Berrah A.; AlSayed M.; Broomfield A.; Donald A.; seif El Dein H.M.; Freisens S.; WUH-LIANG HWU; Peterschmitt M.J.; Yoo H.-W.; Abdelwahab M.
臺大學術典藏 2020-12-16T02:25:49Z Miglustat in patients with Niemann-Pick disease Type C (NP-C): A multicenter observational retrospective cohort study Pineda M.;Wraith J.E.;Mengel E.;Sedel F.;Wuh-Liang Hwu;Rohrbach M.;Bembi B.;Walterfang M.;Korenke G.C.;Marquardt T.;Luzy C.;Giorgino R.;Patterson M.C.; Pineda M.; Wraith J.E.; Mengel E.; Sedel F.; WUH-LIANG HWU; Rohrbach M.; Bembi B.; Walterfang M.; Korenke G.C.; Marquardt T.; Luzy C.; Giorgino R.; Patterson M.C.
臺大學術典藏 2020-12-16T02:25:49Z Miglustat in patients with Niemann-Pick disease Type C (NP-C): A multicenter observational retrospective cohort study Pineda M.;Wraith J.E.;Mengel E.;Sedel F.;Wuh-Liang Hwu;Rohrbach M.;Bembi B.;Walterfang M.;Korenke G.C.;Marquardt T.;Luzy C.;Giorgino R.;Patterson M.C.; Pineda M.; Wraith J.E.; Mengel E.; Sedel F.; WUH-LIANG HWU; Rohrbach M.; Bembi B.; Walterfang M.; Korenke G.C.; Marquardt T.; Luzy C.; Giorgino R.; Patterson M.C.
臺大學術典藏 2020-12-16T02:25:08Z Recommendations for clinical monitoring of patients with acid sphingomyelinase deficiency (ASMD) Wasserstein M.;Dionisi-Vici C.;Giugliani R.;Wuh-Liang Hwu;Lidove O.;Lukacs Z.;Mengel E.;Mistry P.K.;Schuchman E.H.;Mcgovern M.; Wasserstein M.; Dionisi-Vici C.; Giugliani R.; WUH-LIANG HWU; Lidove O.; Lukacs Z.; Mengel E.; Mistry P.K.; Schuchman E.H.; McGovern M.
臺大學術典藏 2020-12-16T02:25:08Z Recommendations for clinical monitoring of patients with acid sphingomyelinase deficiency (ASMD) Wasserstein M.;Dionisi-Vici C.;Giugliani R.;Wuh-Liang Hwu;Lidove O.;Lukacs Z.;Mengel E.;Mistry P.K.;Schuchman E.H.;Mcgovern M.; Wasserstein M.; Dionisi-Vici C.; Giugliani R.; WUH-LIANG HWU; Lidove O.; Lukacs Z.; Mengel E.; Mistry P.K.; Schuchman E.H.; McGovern M.

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