Results 141 to 150 of about 7,801 (251)

Hematopoietic Stem-Cell Gene Therapy for Cystinosis. [PDF]

N Engl J Med
Barshop BA, Ball ED, Benador N, Trauner D, Phillips S, Dohil R, Afshari NA, Roy S, Campo Fernandes B, Kohn D, Shayan K, Everett JK, Bushman FD, Midgley J, Liang H, Sawyers A, Gangoiti JA, Panchal M, Ahmed I, Cherqui S.   +19 more
europepmc   +1 more source

Nephropathic cystinosis: an international consensus document [PDF]

, 2014
Francesco Emma, Galina Nesterova, Craig B. Langman, Alain Labbé, Stéphanie Cherqui, Paul Goodyer, Mirian C. H. Janssen, Marcella Greco, Rezan Topaloğlu, Ewa Elenberg, Ranjan Dohil, Doris A. Trauner, Corinne Antignac, P Cochat, Frederick J. Kaskel, Aude Servais, Elke Wühl, Patrick Niaudet, W. van 't Hoff, WilliamA. Gahl, Elena Levtchenko   +20 more
openalex   +1 more source

Examination of corneal deposits in nephropathic cystinosis using in vivo confocal microscopy and anterior segment optical coherence tomography: an age-dependent cross sectional study [PDF]

, 2020
Anita Csorba, Erika Maka, Otto Alexander Maneschg, Attila J. Szabó, Nóra Szentmáry, Mária Csidey, Miklós Resch, László Imre, Krisztina Knézy, Zoltán Zsolt Nagy   +9 more
openalex   +1 more source

Recent advances in the treatment of cystinosis

, 1995
Jerry A. Schneider, Karen F Clark, A A Greene, Joan Reisch, Thomas C. Markello, William A. Gahl, Jess G. Thoene, Patrick K. Noonan, Ken Berry   +8 more
openalex   +2 more sources

Improving Lifelong Comprehensive Care Coordination in Nephropathic Cystinosis: Multidisciplinary Perspectives. [PDF]

Kidney Int Rep
Golestaneh L, Ames EG, Doyle MH, Ghossein C, Grimm PC, Hewlett JL, Lim A, Marzinelli LS, Schmidt KJ, Smeryage B, Zaritsky JJ, Kaskel FJ.   +11 more
europepmc   +1 more source

Hirschsprung′s disease with infantile nephropathic cystinosis

, 2015
Veereshwar Bhatnagar, Deepak Mittal, Arvind Bagga, Radhika Tandon, MeharChand Sharma   +4 more
openalex   +1 more source

493. Mechanism of Hematopoietic Stem Cell-Mediated Therapy in Cystinosis [PDF]

, 2013

openalex   +1 more source

Cystinosis [PDF]

, 2020
openaire   +3 more sources

Ultrasound Biomicroscopy of the Eye in Cystinosis [PDF]

, 2000
Nils Mungan
openalex   +1 more source

Potential clinical indications for a CCK2 receptor antagonist [PDF]

, 2016
Boyce, Malcolm, Lloyd, Katie A, Pritchard, D Mark   +2 more
core   +1 more source