Pretraga

Rezultati

Long Term Bone Marrow Responses, as Measured by Quantitative Chemical Shift Imaging (QCSI) MRI, Following Treatment With Taliglucerase Alfa in Patients with Type 1 Gaucher Disease [2012]

van, Dussen Laura; Hollak, Carla EM; Zimran, Ari; Petakov, Milan S; Rosenbaum, Hanna; Aviezer, David; Brill-Almon, Einat; Chertkoff, Raul; Maas, Mario

Long-term efficacy and safety results of taliglucerase alfa through 5 years in adult treatment-naive patients with Gaucher disease [2016]

Chertkoff, Raul; Zimran, Ari; Duran, Gloria; Giraldo, Pilar; Rosenbaum, Hanna; Giona, Fiorina; Petakov, Milan S; Eduardo, Solorio-Meza Sergio; Cooper, Peter A; Varughese, Sheeba;

Alon, Sari; Brill-Almon, Einat;

Severe adenylosuccinate lyase deficiency with early autonomic dysfunction: functional characterization of a novel ADSL variant and exploratory treatment with disulfiram [2026]

Kravljanac, Ruzica; Zikanova, Marie; Milacic, Iva; Vucetic-Tadic, Biljana; Popovic, Sofija; Oparnica, Vladimir; Skopova, Vaclava; Forejtova, Alena; Baresova, Veronika

Long-term safety and efficacy data of taliglucerase alfa, a Plant cell-expressed recombinant glucocerebrosidase, in the treatment of naive Gaucher disease patients: 36-Month Results [2013]

Zimran, Ari; Mehta, Atul; Giraldo, Pilar; Rosenbaum, Hanna; Giona, Fiorina; Amato, Dominick J; Petakov, Milan S; Terreros, Munoz Eduardo; Eduardo, Solorio-Meza Sergio; Duran, Gloria;

Elstein, Deborah; Chertkoff, Raul; Brill-Almon, Einat;

Strategies to shorten diagnostic delays for late infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease) [2020]

Mazurkiewicz-Beldinska, Maria; Del Toro, Mireia; Hahn, Andreas; Haliloglu, Goknur; Huidekop, Hidde; Kravljanac, Ružica ; Muhlhausen, Chris; Andersen, Brian Nauheimer; Prpić, Igor; Striano, Pasquale;

Auvin, Stephane;

New transcriptional regulatory element within the intron of phenylalanine hydroxylase gene [2009]

Stojiljković, Maja ; Zukić, Branka ; Tošić, Nataša ; Karan-Đurašević, Teodora ; Radmilović, M. ; Spasovski, Vesna ; Pavlović, Sonja

Significant, continuous improvement in Bone Mineral Density (BMD) among type 1 Gaucher disease patients treated with Velaglucerase Alfa: 69-month experience, including dose reduction [2011]

Elstein, Deborah; Foldes, Joseph A; Zahrieh, David; Cohn, Gabriel M; Djordjevic, Maja S; Brutaru, Costin; Zimran, An

Long Term Safety and Efficacy Data of Taliglucerase Alfa, a Plant Cell Expressed Recombinant Glucocerebrosidase, in Treatment Of Naive Gaucher Disease Patients [2012]

Zimran, Ari; Heitner, Rene; Mehta, Atul; Giraldo, Pllar; Rosenbaum, Hanna; Giona, Florina; Amato, Dominick J; Petakov, Milan S; Meillon, Garcia Luis Antonio; Solorio, Meza Sergio Eduardo;

Duran, Gloria; Elstein, Deborah; Brill-Almon, Einat;

Decreased plasma L-arginine levels in organic acidurias (MMA and PA) and decreased plasma branched-chain amino acid levels in urea cycle disorders as a potential cause of growth retardation: Options for treatment [2019]

Molema, Femke; ...; Đorđević, Maja S. ; Sarajlija, Adrijan ; ...; (broj koautora 67)

Novel transcriptional regulatory element in the phenylalanine hydroxylase gene intron 8 [2010]

Stojiljković, Maja ; Zukić, Branka ; Tošić, Nataša ; Karan-Đurašević, Teodora ; Spasovski, Vesna ; Nikčević, Gordana ; Pavlović, Sonja