The New England journal of medicine, 2020-06, Vol.382 (24), p.2289-2301
- Balwani, Manisha
- Sardh, Eliane
- Ventura, Paolo
- Peiró, Paula Aguilera
- Rees, David C
- Stölzel, Ulrich
- Bissell, D. Montgomery
- Bonkovsky, Herbert L
- Windyga, Jerzy
- Anderson, Karl E
- Parker, Charles
- Silver, Samuel M
- Keel, Siobán B
- Wang, Jiaan-Der
- Stein, Penelope E
- Harper, Pauline
- Vassiliou, Daphne
- Wang, Bruce
- Phillips, John
- Ivanova, Aneta
- Langendonk, Janneke G
- Kauppinen, Raili
- Minder, Elisabeth
- Horie, Yutaka
- Penz, Craig
- Chen, Jihong
- Liu, Shangbin
- Ko, John J
- Sweetser, Marianne T
- Garg, Pushkal
- Vaishnaw, Akshay
- Kim, Jae B
- Simon, Amy R
- Gouya, Laurent
2020
Details
- Autor(en) / Beteiligte
- Balwani, Manisha
- Sardh, Eliane
- Ventura, Paolo
- Peiró, Paula Aguilera
- Rees, David C
- Stölzel, Ulrich
- Bissell, D. Montgomery
- Bonkovsky, Herbert L
- Windyga, Jerzy
- Anderson, Karl E
- Parker, Charles
- Silver, Samuel M
- Keel, Siobán B
- Wang, Jiaan-Der
- Stein, Penelope E
- Harper, Pauline
- Vassiliou, Daphne
- Wang, Bruce
- Phillips, John
- Ivanova, Aneta
- Langendonk, Janneke G
- Kauppinen, Raili
- Minder, Elisabeth
- Horie, Yutaka
- Penz, Craig
- Chen, Jihong
- Liu, Shangbin
- Ko, John J
- Sweetser, Marianne T
- Garg, Pushkal
- Vaishnaw, Akshay
- Kim, Jae B
- Simon, Amy R
- Gouya, Laurent
- Titel
- Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria
- Ist Teil von
- The New England journal of medicine, 2020-06, Vol.382 (24), p.2289-2301
- Ort / Verlag
- Boston: Massachusetts Medical Society
- Erscheinungsjahr
- 2020
- Link zum Volltext
- Quelle
- EZB-FREE-00999 freely available EZB journals
- Beschreibungen/Notizen
- In a randomized phase 3 trial involving patients with acute intermittent porphyria, the use of givosiran, an oligonucleotide drug designed to target messenger RNA encoding aminolevulinic acid synthase, led to a 74% lower annualized porphyria attack rate than the use of placebo at 6 months.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0028-4793, 1533-4406eISSN: 1533-4406DOI: 10.1056/NEJMoa1913147Titel-ID: cdi_swepub_primary_oai_prod_swepub_kib_ki_se_143941324
- Format
- –
- Schlagworte
- Aminolevulinic acid, Body weight, Chronic illnesses, Complications and side effects, Creatinine, Drug therapy, Glomerular filtration rate, Hemin, Hypertension, Intravenous administration, Liver, Liver diseases, Medicin och hälsovetenskap, Mutation, Pain, Patient outcomes, Patients, Porphyria, RNA-mediated interference, Statistical analysis