The New England journal of medicine, 2021-04, Vol.384 (13), p.1216-1226
- Garrelfs, Sander F
- Frishberg, Yaacov
- Hulton, Sally A
- Koren, Michael J
- O’Riordan, William D
- Cochat, Pierre
- Deschênes, Georges
- Shasha-Lavsky, Hadas
- Saland, Jeffrey M
- van’t Hoff, William G
- Fuster, Daniel G
- Magen, Daniella
- Moochhala, Shabbir H
- Schalk, Gesa
- Simkova, Eva
- Groothoff, Jaap W
- Sas, David J
- Meliambro, Kristin A
- Lu, Jiandong
- Sweetser, Marianne T
- Garg, Pushkal P
- Vaishnaw, Akshay K
- Gansner, John M
- McGregor, Tracy L
- Lieske, John C
2021
Details
- Autor(en) / Beteiligte
- Garrelfs, Sander F
- Frishberg, Yaacov
- Hulton, Sally A
- Koren, Michael J
- O’Riordan, William D
- Cochat, Pierre
- Deschênes, Georges
- Shasha-Lavsky, Hadas
- Saland, Jeffrey M
- van’t Hoff, William G
- Fuster, Daniel G
- Magen, Daniella
- Moochhala, Shabbir H
- Schalk, Gesa
- Simkova, Eva
- Groothoff, Jaap W
- Sas, David J
- Meliambro, Kristin A
- Lu, Jiandong
- Sweetser, Marianne T
- Garg, Pushkal P
- Vaishnaw, Akshay K
- Gansner, John M
- McGregor, Tracy L
- Lieske, John C
- Titel
- Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1
- Ist Teil von
- The New England journal of medicine, 2021-04, Vol.384 (13), p.1216-1226
- Ort / Verlag
- United States: Massachusetts Medical Society
- Erscheinungsjahr
- 2021
- Link zum Volltext
- Quelle
- MEDLINE
- Beschreibungen/Notizen
- Primary hyperoxaluria type 1 is caused by hepatic overproduction of oxalate, leading to kidney stones, nephrocalcinosis, kidney failure, and systemic oxalosis. This trial tested whether an oligonucleotide drug can reduce the production of hepatic oxalate.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0028-4793eISSN: 1533-4406DOI: 10.1056/NEJMoa2021712Titel-ID: cdi_proquest_miscellaneous_2507731776
- Format
- –
- Schlagworte
- Adolescent, Adult, Age, Calcinosis, Care and treatment, Child, Complications and side effects, Creatinine, Creatinine - urine, Demographic aspects, Development and progression, Double-Blind Method, Enzymes, Excretion, Female, Glomerular Filtration Rate, Humans, Hyperoxaluria, Hyperoxaluria, Primary - blood, Hyperoxaluria, Primary - complications, Hyperoxaluria, Primary - drug therapy, Hyperoxaluria, Primary - urine, Kidney Calculi - prevention & control, Kidney diseases, Kidney failure, Kidney stones, Laboratories, Liver, Liver diseases, Male, Metabolism, Middle Aged, Nephrolithiasis, Ostomy, Oxalates - blood, Oxalates - metabolism, Oxalates - urine, Oxalic acid, Oxalosis, Oxaluria, Placebos, Plasma, Primary hyperoxaluria, Renal failure, Risk factors, RNA, Small Interfering - adverse effects, RNA, Small Interfering - therapeutic use, RNA-mediated interference, RNAi Therapeutics, Statistical analysis, Young Adult