Page last updated: 2024-08-23

acetylgalactosamine and Porphyria, Acute Intermittent

acetylgalactosamine has been researched along with Porphyria, Acute Intermittent in 19 studies

Research

Studies (19)

TimeframeStudies, this research(%)All Research%
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's0 (0.00)29.6817
2010's1 (5.26)24.3611
2020's18 (94.74)2.80

Authors

AuthorsStudies
Aguilera-Peiró, P; Anderson, DKE; Balwani, M; Bonkovsky, HL; Gouya, L; Hua, Z; Ko, JJ; Kuter, DJ; Monroy, S; Montgomery Bissell, D; Oh, J; Parker, C; Ritchie, B; Sardh, E; Stein, PE; Sweetser, MT; Ventura, P1
Bouchoule, I; Cerutti, D; Douillard, C; Duchêne, F; Dupré, T; Faivre, A; Fiorentino, V; Gouya, L; Grobost, V; Lefebvre, T; Manceau, H; Mirmiran, A; Moulouel, B; Peoc'h, K; Poli, A; Puy, H; Rivière, S; Schmitt, C; Talbi, N1
Harper, P; Sardh, E1
Balado González, AM; Feijoo-Massó, C; Navarro-Saez, MDC1
de Paula Brandão, PR; Titze-de-Almeida, R; Titze-de-Almeida, SS1
Agarwal, S; Clausen, VA; Goel, V; Habtemariam, BA; Kim, JB; Robbie, GJ; Simon, AR1
Anderson, KE; Balwani, M; Bissell, DM; Bonkovsky, HL; Chen, J; Garg, P; Gouya, L; Harper, P; Horie, Y; Ivanova, A; Kauppinen, R; Keel, SB; Kim, JB; Ko, JJ; Langendonk, JG; Liu, S; Minder, E; Parker, C; Peiró, PA; Penz, C; Phillips, J; Rees, DC; Sardh, E; Silver, SM; Simon, AR; Stein, PE; Stölzel, U; Sweetser, MT; Vaishnaw, A; Vassiliou, D; Ventura, P; Wang, B; Wang, JD; Windyga, J1
Gonzalez-Aseguinolaza, G1
Stölzel, U1
Bonkovsky, HL; Epstein, J; Hurd, J; Massachi, S1
Gomá-Garcés, E; Ortíz, A; Pérez-Gómez, MV1
Balwani, M; Gouya, L; Sardh, E1
Fuchs, K; Hanyk, K; Kilic, M; Linker, RA; Neumann, B; Schlachetzki, F; Steinberg, T1
Bonkovsky, HL; Honor, A; Rudnick, SR1
Aarsand, AK; Aguilera, P; Brunet, M; Deulofeu, R; García-Villoria, J; Gómez-Gómez, À; Pozo, OJ; Sandberg, S; To-Figueras, J; Wijngaard, R1
Syed, YY1
Beykirch, MK; Klein, M; Loehr, C; Molitor, B; Obermeier, Z; Petrides, PE; Schuhmann, E; Torkler, H1
Arranz, E; Ávila, MA; Enríquez de Salamanca, R; Fontanellas, A; Morales-Conejo, M1
Anderson, KE; Balwani, M; Bissell, DM; Bonkovsky, HL; Chan-Daniels, A; Desnick, R; Fitzgerald, K; Garg, P; Harper, P; He, Q; Kim, JB; Parker, C; Penz, C; Phillips, J; Querbes, W; Rees, D; Sardh, E; Simon, AR; Stein, P; Vaishnaw, A; Vassiliou, D1

Reviews

3 review(s) available for acetylgalactosamine and Porphyria, Acute Intermittent

ArticleYear
RNAi therapy with givosiran significantly reduces attack rates in acute intermittent porphyria.
    Journal of internal medicine, 2022, Volume: 291, Issue:5

    Topics: Acetylgalactosamine; Adolescent; Adult; Heme; Humans; Incidence; Porphobilinogen Synthase; Porphyria, Acute Intermittent; Porphyrias, Hepatic; Pyrrolidines; Quality of Life; RNAi Therapeutics

2022
Leading RNA Interference Therapeutics Part 2: Silencing Delta-Aminolevulinic Acid Synthase 1, with a Focus on Givosiran.
    Molecular diagnosis & therapy, 2020, Volume: 24, Issue:1

    Topics: Acetylgalactosamine; Clinical Trials as Topic; Drug Development; Gene Silencing; Genetic Therapy; Heme; Humans; Nitric Oxide Synthase Type I; Porphyria, Acute Intermittent; Pyrrolidines; RNA Interference; RNA, Small Interfering; Treatment Outcome

2020
Givosiran: A Review in Acute Hepatic Porphyria.
    Drugs, 2021, Volume: 81, Issue:7

    Topics: 5-Aminolevulinate Synthetase; Acetylgalactosamine; Acute Kidney Injury; Chemical and Drug Induced Liver Injury; Drug Interactions; Hemin; Hospitalization; Humans; Pain; Porphobilinogen Synthase; Porphyria, Acute Intermittent; Porphyrias, Hepatic; Pyrrolidines; Randomized Controlled Trials as Topic; RNA, Small Interfering; Severity of Illness Index

2021

Trials

5 trial(s) available for acetylgalactosamine and Porphyria, Acute Intermittent

ArticleYear
Efficacy and safety of givosiran for acute hepatic porphyria: 24-month interim analysis of the randomized phase 3 ENVISION study.
    Liver international : official journal of the International Association for the Study of the Liver, 2022, Volume: 42, Issue:1

    Topics: Acetylgalactosamine; Humans; Porphyria, Acute Intermittent; Porphyrias, Hepatic; Pyrrolidines; Quality of Life

2022
Pharmacokinetics and Pharmacodynamics of the Small Interfering Ribonucleic Acid, Givosiran, in Patients With Acute Hepatic Porphyria.
    Clinical pharmacology and therapeutics, 2020, Volume: 108, Issue:1

    Topics: Acetylgalactosamine; Adult; Aminolevulinic Acid; Dose-Response Relationship, Drug; Drug Administration Schedule; Female; Half-Life; Humans; Injections, Subcutaneous; Male; Middle Aged; Porphobilinogen; Porphyria, Acute Intermittent; Pyrrolidines; Young Adult

2020
Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria.
    The New England journal of medicine, 2020, 06-11, Volume: 382, Issue:24

    Topics: Acetylgalactosamine; Adult; Aminolevulinic Acid; Double-Blind Method; Fatigue; Female; Humans; Injections, Subcutaneous; Least-Squares Analysis; Liver; Male; Nausea; Pain; Patient Outcome Assessment; Porphobilinogen; Porphyria, Acute Intermittent; Pyrrolidines; Renal Insufficiency, Chronic; RNAi Therapeutics; Transaminases

2020
[Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria].
    Zeitschrift fur Gastroenterologie, 2020, Volume: 58, Issue:8

    Topics: Acetylgalactosamine; Humans; Molecular Targeted Therapy; Porphyria, Acute Intermittent; Pyrrolidines; RNAi Therapeutics; Treatment Outcome

2020
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria.
    The New England journal of medicine, 2019, 02-07, Volume: 380, Issue:6

    Topics: 5-Aminolevulinate Synthetase; Acetylgalactosamine; Adult; Amides; Dose-Response Relationship, Drug; Drug Administration Schedule; Female; Humans; Injections, Subcutaneous; Liver; Male; Middle Aged; Molecular Targeted Therapy; Porphobilinogen; Porphyria, Acute Intermittent; Pyrrolidines; RNA, Messenger; RNAi Therapeutics

2019

Other Studies

11 other study(ies) available for acetylgalactosamine and Porphyria, Acute Intermittent

ArticleYear
Givosiran in acute intermittent porphyria: A personalized medicine approach.
    Molecular genetics and metabolism, 2022, Volume: 135, Issue:3

    Topics: Acetylgalactosamine; Acute Disease; Heme; Humans; Pancreatitis; Porphyria, Acute Intermittent; Precision Medicine; Pyrrolidines; Retrospective Studies

2022
Acute intermittent porphyria: Acute flare-up resolved with givosiran.
    Medicina clinica, 2023, Nov-24, Volume: 161, Issue:10

    Topics: Acetylgalactosamine; Humans; Porphyria, Acute Intermittent; Pyrrolidines

2023
Givosiran - Running RNA Interference to Fight Porphyria Attacks.
    The New England journal of medicine, 2020, 06-11, Volume: 382, Issue:24

    Topics: Acetylgalactosamine; Humans; Porphyria, Acute Intermittent; Porphyrias; Pyrrolidines; RNA Interference; RNAi Therapeutics; Running

2020
Cost savings with hemin versus givosiran for the treatment of patients with acute intermittent porphyria (AIP).
    Journal of medical economics, 2020, Volume: 23, Issue:12

    Topics: Acetylgalactosamine; Adult; Cost Savings; Hemin; Humans; Porphyria, Acute Intermittent; Pyrrolidines

2020
Givosiran for Acute Intermittent Porphyria.
    The New England journal of medicine, 2020, 11-12, Volume: 383, Issue:20

    Topics: Acetylgalactosamine; Amides; Humans; Porphyria, Acute Intermittent; Pyrrolidines; RNA Interference; RNAi Therapeutics

2020
Givosiran for Acute Intermittent Porphyria. Reply.
    The New England journal of medicine, 2020, 11-12, Volume: 383, Issue:20

    Topics: Acetylgalactosamine; Amides; Humans; Porphyria, Acute Intermittent; Pyrrolidines

2020
Case report of a complicated neurologically manifesting acute porphyria treated successfully with Givosiran.
    Journal of the neurological sciences, 2021, 03-15, Volume: 422

    Topics: Acetylgalactosamine; Humans; Porphyria, Acute Intermittent; Pyrrolidines

2021
Givosiran to treat acute porphyria.
    Drugs of today (Barcelona, Spain : 1998), 2021, Volume: 57, Issue:1

    Topics: Acetylgalactosamine; Adolescent; Adult; Child; Humans; Porphyria, Acute Intermittent; Porphyrias, Hepatic; Pyrrolidines; United States

2021
Dysregulation of homocysteine homeostasis in acute intermittent porphyria patients receiving heme arginate or givosiran.
    Journal of inherited metabolic disease, 2021, Volume: 44, Issue:4

    Topics: Acetylgalactosamine; Adult; Arginine; Cystathionine beta-Synthase; Female; Folic Acid; Heme; Homeostasis; Homocysteine; Homocystinuria; Humans; Hydroxymethylbilane Synthase; Hyperhomocysteinemia; Male; Methionine; Middle Aged; Porphyria, Acute Intermittent; Pyridoxal Phosphate; Pyrrolidines; Young Adult

2021
Severe homocysteinemia in two givosiran-treated porphyria patients: is free heme deficiency the culprit?
    Annals of hematology, 2021, Volume: 100, Issue:7

    Topics: 5-Aminolevulinate Synthetase; Acetylgalactosamine; Adult; Arginine; Colitis; Colon, Sigmoid; Controlled Clinical Trials as Topic; Drug Hypersensitivity; Female; Fibrosis; Heme; Hepatocytes; High-Throughput Nucleotide Sequencing; Homocysteine; Humans; Hydroxymethylbilane Synthase; Hyperhomocysteinemia; Male; Models, Biological; Pancreatitis; Porphyria, Acute Intermittent; Pyrrolidines

2021
Acute intermittent porphyria, givosiran, and homocysteine.
    Journal of inherited metabolic disease, 2021, Volume: 44, Issue:4

    Topics: Acetylgalactosamine; Homocysteine; Humans; Porphyria, Acute Intermittent; Pyrrolidines

2021