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Safety and efficacy of the terminal complement inhibitor eculizumab in Japanese patients with paroxysmal nocturnal hemoglobinuria: the AEGIS clinical trial.
Published in:
2011
By:
- Kanakura, Yuzuru;
- Ohyashiki, Kazuma;
- Shichishima, Tsutomu;
- Okamoto, Shinichiro;
- Ando, Kiyoshi;
- Ninomiya, Haruhiko;
- Kawaguchi, Tatsuya;
- Nakao, Shinji;
- Nakakuma, Hideki;
- Nishimura, Jun-ichi;
- Kinoshita, Taroh;
- Bedrosian, Camille;
- Valentine, Marye;
- Khursigara, Gus;
- Ozawa, Keiya;
- Omine, Mitsuhiro;
- Bedrosian, Camille L;
- Valentine, Marye Ellen
Publication type:
journal article
A Reference Range for Plasma Levels of Inorganic Pyrophosphate in Children Using the ATP Sulfurylase Method.
Published in:
Journal of Clinical Endocrinology & Metabolism, 2022, v. 107, n. 1, p. 109, doi. 10.1210/clinem/dgab615
By:
- Bernhard, Eva;
- Nitschke, Yvonne;
- Khursigara, Gus;
- Sabbagh, Yves;
- Yongbao Wang;
- Rutsch, Frank
Publication type:
Article
Response of enthesopathy in ENPP1 deficiency to enzyme replacement therapy in murine models and enthesopathy comorbidities and quality of life in ENPP1‐deficient adults.
Published in:
FASEB Journal, 2022, v. 36, p. N.PAG, doi. 10.1096/fasebj.2022.36.S1.R5311
By:
- Ansh, Anenya J.;
- Nester, Catherine;
- O'Brien, Christine;
- Stabach, Paul R.;
- Murtada, Sae‐Il;
- Lester, Ethan R.;
- Khursigara, Gus;
- Molloy, Liz;
- Carpenter, Thomas O.;
- Ferreira, Carlos R.;
- Braddock, Demetrios T.
Publication type:
Article
p75 neurotrophin receptor as a modulator of survival and death decisions.
Published in:
Microscopy Research & Technique, 1999, v. 45, n. 4/5, p. 217, doi. 10.1002/(SICI)1097-0029(19990515/01)45:4/5<217::AID-JEMT5>3.0.CO;2-5
By:
- Casaccia-Bonnefil, Patrizia;
- Gu, Chenghua;
- Khursigara, Gus;
- Chao, Moses V.
Publication type:
Article
Erratum: 'Long-term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinura' by Hillmen et al., Am J Hematol 553-559, 2010, DOI number 21757.
Published in:
American Journal of Hematology, 2010, v. 85, n. 11, p. 911, doi. 10.1002/ajh.21861
By:
- Hillmen, Peter;
- Elebute, Modupe;
- Kelly, Richard;
- Urbano-Ispizua, Alvaro;
- Hill, Anita;
- Rother, Russell P.;
- Khursigara, Gus;
- Fu, Chieh-Lin;
- Omine, Mitsuhiro;
- Browne, Paul;
- Rosse, Wendell
Publication type:
Article
Long-term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinuria.
Published in:
American Journal of Hematology, 2010, v. 85, n. 8, p. 553, doi. 10.1002/ajh.21757
By:
- Hillmen, Peter;
- Elebute, Modupe;
- Kelly, Richard;
- Urbano-Ispizua, Alvaro;
- Hill, Anita;
- Rother, Russell P.;
- Khursigara, Gus;
- Fu, Chieh-Lin;
- Omine, Mitsuhiro;
- Browne, Paul;
- Rosse, Wendell
Publication type:
Article
Lifelong impact of ENPP1 Deficiency and the early onset form of ABCC6 Deficiency from patient or caregiver perspective.
Published in:
PLoS ONE, 2022, v. 17, n. 7, p. 1, doi. 10.1371/journal.pone.0270632
By:
- O'Brien, Christine;
- Khursigara, Gus;
- Huertas, Pedro;
- Leiro, Beth;
- Molloy, Liz;
- Nester, Catherine
Publication type:
Article
The management of pregnancy in paroxysmal nocturnal haemoglobinuria on long term eculizumab.
Published in:
British Journal of Haematology, 2010, v. 149, n. 3, p. 446, doi. 10.1111/j.1365-2141.2010.08099.x
By:
- Kelly, Richard;
- Arnold, Louise;
- Richards, Stephen;
- Hill, Anita;
- Bomken, Charlotte;
- Hanley, John;
- Loughney, Andrew;
- Beauchamp, Jon;
- Khursigara, Gus;
- Rother, Russell P.;
- Chalmers, Elizabeth;
- Fyfe, Andrew;
- Fitzsimons, Edward;
- Nakamura, Ryotaro;
- Gaya, Anna;
- Risitano, Antonio M.;
- Schubert, Jörg;
- Norfolk, Derek;
- Simpson, Nigel;
- Hillmen, Peter
Publication type:
Article
ENPP1 deficiency: A clinical update on the relevance of individual variants using a locus‐specific patient database.
Published in:
Human Mutation, 2022, v. 43, n. 12, p. 1673, doi. 10.1002/humu.24477
By:
- Mercurio, Stephanie A.;
- Chunn, Lauren M.;
- Khursigara, Gus;
- Nester, Catherine;
- Wray, Kathleen;
- Botschen, Ulrike;
- Kiel, Mark J.;
- Rutsch, Frank;
- Ferreira, Carlos R.
Publication type:
Article
Musculoskeletal Comorbidities and Quality of Life in ENPP1‐Deficient Adults and the Response of Enthesopathy to Enzyme Replacement Therapy in Murine Models.
Published in:
Journal of Bone & Mineral Research, 2022, v. 37, n. 3, p. 494, doi. 10.1002/jbmr.4487
By:
- Ferreira, Carlos R.;
- Ansh, Anenya Jai;
- Nester, Catherine;
- O'Brien, Christine;
- Stabach, Paul R.;
- Murtada, Sae‐Il;
- Lester, Ethan R.;
- Khursigara, Gus;
- Molloy, Liz;
- Carpenter, Thomas O.;
- Braddock, Demetrios T.
Publication type:
Article
Ectopic Calcification and Hypophosphatemic Rickets: Natural History of ENPP1 and ABCC6 Deficiencies.
Published in:
Journal of Bone & Mineral Research, 2021, v. 36, n. 11, p. 2193, doi. 10.1002/jbmr.4418
By:
- Ferreira, Carlos R;
- Kintzinger, Kristina;
- Hackbarth, Mary E;
- Botschen, Ulrike;
- Nitschke, Yvonne;
- Mughal, M Zulf;
- Baujat, Genevieve;
- Schnabel, Dirk;
- Yuen, Eric;
- Gahl, William A;
- Gafni, Rachel I;
- Liu, Qing;
- Huertas, Pedro;
- Khursigara, Gus;
- Rutsch, Frank
Publication type:
Article

Found: 11