Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 30-month analysis of the phase 3 ILLUMINATE-B trial.

Autor: Frishberg Y; Division of Pediatric Nephrology, Shaare Zedek Medical Center and Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel., Hayes W; Department of Paediatric Nephrology, Great Ormond Street Hospital, London, United Kingdom., Shasha-Lavsky H; Pediatric Nephrology Unit, Galilee Medical Center, Nahariya, Israel.; Azrieli Faculty of Medicine, Bar Ilan University, Safed, Israel., Sas DJ; Division of Pediatric Nephrology and Hypertension, Mayo Clinic, Rochester, MN, United States., Michael M; Division of Pediatric Nephrology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX, United States., Sellier-Leclerc AL; Hôpital Femme Mère Enfant and Centre d'Investigation Clinique Inserm, Hospices Civils de Lyon, ERKnet, Bron, France., Hogan J; Pediatric Nephrology Department, Hôpital Robert-Debré, APHP, Paris, France., Willey R; Biostatistics, Alnylam Pharmaceuticals, Cambridge, MA, United States., Gansner JM; Clinical Development, Alnylam Pharmaceuticals, Cambridge, MA, United States., Magen D; Pediatric Nephrology Institute, Rambam Health Care Campus, and Faculty of Medicine, Technion - Israel Institute of Technology, Haifa, Israel.

Publikováno v: Frontiers in pediatrics [Front Pediatr] 2024 Sep 16; Vol. 12, pp. 1392644. Date of Electronic Publication: 2024 Sep 16 (Print Publication: 2024).

Quantification of oxalate by novel LC-MS/MS: assay development, validation and application in lumasiran clinical trials.

Autor: Clausen VA; Alnylam Pharmaceuticals, Cambridge, MA 02142, USA., Cao KH; Alnylam Pharmaceuticals, Cambridge, MA 02142, USA., Gansner JM; Alnylam Pharmaceuticals, Cambridge, MA 02142, USA., Robbie GJ; Alnylam Pharmaceuticals, Cambridge, MA 02142, USA., Wu JT; Alnylam Pharmaceuticals, Cambridge, MA 02142, USA.

Publikováno v: Bioanalysis [Bioanalysis] 2023 May; Vol. 15 (9), pp. 481-491. Date of Electronic Publication: 2023 May 17.

Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial.

Autor: Michael M; Division of Pediatric Nephrology, Department of Pediatrics, Texas Children's Hospital/Baylor College of Medicine, Houston, Texas. Electronic address: mmichael@bcm.edu., Groothoff JW; Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands., Shasha-Lavsky H; Pediatric Nephrology Unit, Galilee Medical Center, Azrieli Faculty of Medicine, Bar Ilan University, Nahariya, Israel., Lieske JC; Division of Nephrology and Hypertension, Mayo Clinic, Rochester, Minnesota., Frishberg Y; Division of Pediatric Nephrology, Shaare Zedek Medical Center, Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel., Simkova E; Nephrology - Medical Affairs, Al Jalila Children's Hospital, Dubai, United Arab Emirates., Sellier-Leclerc AL; Hôpital Femme Mère Enfant en Centre d'Investigation Clinique, Institut National de la Santé et de la Recherche Médicale (INSERM), Hospices Civils de Lyon, ERKnet, Bron, France., Devresse A; Division of Nephrology, Cliniques Universitaires Saint-Luc, Brussels, Belgium., Guebre-Egziabher F; Nephrology and Renal Function Unit, Edouard Herriot Hospital, Hospices Civils de Lyon, INSERM 1060, Lyon, France., Bakkaloglu SA; Department of Pediatric Nephrology, Faculty of Medicine, Gazi University, Ankara, Turkey., Mourani C; Department of Pediatrics, Hôtel-Dieu de France Hospital, Beirut, Lebanon., Saqan R; Pharmaceutical Research Center, Jordan University of Science and Technology, Irbid, Jordan., Singer R; Renal Service, Canberra Health Services, Garran, ACT, Australia., Willey R; Alnylam Pharmaceuticals, Cambridge, Massachusetts., Habtemariam B; Alnylam Pharmaceuticals, Cambridge, Massachusetts., Gansner JM; Alnylam Pharmaceuticals, Cambridge, Massachusetts., Bhan I; Alnylam Pharmaceuticals, Cambridge, Massachusetts., McGregor T; Alnylam Pharmaceuticals, Cambridge, Massachusetts., Magen D; Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa, Israel.

Publikováno v: American journal of kidney diseases : the official journal of the National Kidney Foundation [Am J Kidney Dis] 2023 Feb; Vol. 81 (2), pp. 145-155.e1. Date of Electronic Publication: 2022 Jul 14.

Randomized Clinical Trial on the Long-Term Efficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1.

Autor: Hulton SA; Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham, UK., Groothoff JW; Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands., Frishberg Y; Division of Pediatric Nephrology, Shaare Zedek Medical Center and Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel., Koren MJ; Jacksonville Center for Clinical Research, Jacksonville, Florida, USA., Overcash JS; Velocity Clinical Research, San Diego, California, USA., Sellier-Leclerc AL; Hôpital Femme Mère Enfant and Centre d'Investigation Clinique Institut National de la Santé et de la Recherche Médicale, Hospices Civils de Lyon, ERKnet, Bron, France., Shasha-Lavsky H; Pediatric Nephrology Unit, Galilee Medical Center and Azrieli Faculty of Medicine, Bar-Ilan University, Nahariya, Israel., Saland JM; Icahn School of Medicine at Mount Sinai, New York, New York, USA., Hayes W; Department of Pediatric Nephrology, Great Ormond Street Hospital, London, UK., Magen D; Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa, Israel., Moochhala SH; UCL Department of Renal Medicine, Royal Free Hospital, London, UK., Coenen M; Department of Clinical Chemistry and Clinical Pharmacology, University Hospital Bonn, Bonn, Germany., Simkova E; Al Jalila Children's Hospital, Dubai, United Arabs Emirates., Garrelfs SF; Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands., Sas DJ; Division of Pediatric Nephrology and Hypertension, Mayo Clinic, Rochester, Minnesota, USA., Meliambro KA; Icahn School of Medicine at Mount Sinai, New York, New York, USA., Ngo T; Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA., Sweetser MT; Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA., Habtemariam BA; Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA., Gansner JM; Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA., McGregor TL; Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA., Lieske JC; Division of Nephrology and Hypertension, Mayo Clinic, Rochester, Minnesota, USA.

Publikováno v: Kidney international reports [Kidney Int Rep] 2021 Dec 11; Vol. 7 (3), pp. 494-506. Date of Electronic Publication: 2021 Dec 11 (Print Publication: 2022).

Distinct genetic pathways define pre-malignant versus compensatory clonal hematopoiesis in Shwachman-Diamond syndrome.

Autor: Kennedy AL; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA., Myers KC; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.; Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., Bowman J; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA.; Institute for Protein Innovation, Boston, MA, USA., Gibson CJ; Department of Medical Oncology, Division of Hematological Malignancies Dana-Farber Cancer Institute, Boston, MA, USA., Camarda ND; Dana-Farber Cancer Institute, Boston, MA, USA., Furutani E; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA., Muscato GM; Boston Children's Hospital, Boston, MA, USA., Klein RH; Dana-Farber Cancer Institute, Boston, MA, USA.; Broad Institute, Boston, MA, USA., Ballotti K; Boston Children's Hospital, Boston, MA, USA., Liu S; Biostatistics and Research Design Center, Institutional Centers for Clinical and Translational Research, Boston Children's Hospital, Boston, MA, USA., Harris CE; Boston Children's Hospital, Boston, MA, USA., Galvin A; Boston Children's Hospital, Boston, MA, USA., Malsch M; Boston Children's Hospital, Boston, MA, USA., Dale D; Department of Internal Medicine, University of Washington, Seattle, WA, USA., Gansner JM; Division of Hematology, Department of Medicine, Brigham and Women's Hospital, Boston, MA, USA., Nakano TA; Center for Cancer and Blood Disorders, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO, USA., Bertuch A; Department of Pediatrics/Hematology-Oncology, Baylor College of Medicine, Houston, TX, USA., Vlachos A; Division of Hematology/Oncology and Cellular Therapy, Cohen Children's Medical Center of New York, New Hyde Park, NY, USA.; Zucker School of Medicine at Hofstra/Northwell School of Medicine, Hempstead, NY, USA., Lipton JM; Division of Hematology/Oncology and Cellular Therapy, Cohen Children's Medical Center of New York, New Hyde Park, NY, USA.; Zucker School of Medicine at Hofstra/Northwell School of Medicine, Hempstead, NY, USA., Castillo P; Shands Children's Hospital, Department of Pediatrics, Division of Pediatric Hematology Oncology, University of Florida, Gainesville, FL, USA., Connelly J; Department of Pediatrics, Division of Pediatric Hematology Oncology, Vanderbilt University Medical Center, Nashville, TN, USA., Churpek J; Department of Medicine, Section of Hematology, Oncology, and Palliative Care, The University of Wisconsin-Madison, Madison, WI, USA., Edwards JR; Indiana Blood and Marrow Transplantation, Indianapolis, IN, USA., Hijiya N; Department of Pediatrics, Columbia University Medical Center, New York, NY, USA., Ho RH; Department of Pediatrics, Division of Pediatric Hematology Oncology, Vanderbilt University Medical Center, Nashville, TN, USA., Hofmann I; Department of Pediatrics, Division of Pediatric Hematology/Oncology and BMT, University of Wisconsin, Madison, WI, USA., Huang JN; Department of Pediatrics, UCSF Benioff Children's Hospital, San Francisco, CA, USA.; Division of Pediatric Allergy, Immunology, and Blood & Marrow Transplantation, UCSF Benioff Children's Hospital, San Francisco, CA, USA., Keel S; Division of Hematology, Department of Medicine, University of Washington, Seattle, WA, USA., Lamble A; Division of Hematology-Oncology, Seattle Children's Hospital, Seattle, WA, USA., Lau BW; Dartmouth-Hitchcock Medical Center, Pediatric Hematology Oncology, Geisel School of Medicine, Lebanon, NH, USA.; Department of Medicine, University of Florida, Gainesville, FL, USA., Norkin M; Division of Cancer Medicine, Baptist MD Anderson Cancer Center, Jacksonville, FL, USA., Stieglitz E; Department of Pediatrics, UCSF Benioff Children's Hospital, San Francisco, CA, USA.; UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco, CA, USA., Stock W; Department of Medicine, University of Chicago, Chicago, IL, USA., Walkovich K; Division of Pediatric Hematology- Oncology, Department of Pediatrics, University of Michigan Medical School, Ann Arbor, MI, USA., Boettcher S; Department of Medical Oncology and Hematology, University Hospital Zurich and University of Zurich, Zurich, Switzerland., Brendel C; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA.; Harvard Stem Cell Institute, Cambridge, MA, USA., Fleming MD; Department of Pathology, Boston Children's Hospital, Boston, MA, USA., Davies SM; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.; Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., Weller EA; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.; Biostatistics and Research Design Center, Institutional Centers for Clinical and Translational Research, Boston Children's Hospital, Boston, MA, USA., Bahl C; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA.; Institute for Protein Innovation, Boston, MA, USA., Carter SL; Broad Institute, Boston, MA, USA.; Joint Center for Cancer Precision Medicine, Dana-Farber Cancer Institute/Brigham and Women's Hospital, Boston, MA, USA., Shimamura A; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA., Lindsley RC; Department of Medical Oncology, Division of Hematological Malignancies Dana-Farber Cancer Institute, Boston, MA, USA. Coleman_Lindsley@DFCI.HARVARD.EDU.

Publikováno v: Nature communications [Nat Commun] 2021 Feb 26; Vol. 12 (1), pp. 1334. Date of Electronic Publication: 2021 Feb 26.