Results 141 to 150 of about 1,551 (185)

Understanding Hepatic Porphyrias: Symptoms, Treatments, and Unmet Needs. [PDF]

Semin Liver Dis
Balogun O, Nejak-Bowen K.
europepmc   +1 more source

Design and systematic evaluation of novel chemical modification patterns enable the identification of a potent siRNA targeting <i>PCSK9</i>. [PDF]

Mol Ther Methods Clin Dev
Huang ZA, Yang S, Ji GS, Gao ZC, Li ZM, Wang J, Yu F, Song GS.   +7 more
europepmc   +1 more source

The RNA revolution in medicine: from gene regulation to clinical therapeutics. [PDF]

Anim Cells Syst (Seoul)
Jeong J, Jeong S.
europepmc   +1 more source

Zilebesiran as an Innovative siRNA-Based Therapeutic Approach for Hypertension: Emerging Perspectives in Cardiovascular Medicine. [PDF]

Int J Mol Sci
Morosan PA, Bobu AM, Carauleanu A, Popa R, Costea CF, Filip C, Buzduga CM, Patrascanu E, Cucu AI, Tudosa RI, Covali R, Haisan A.   +11 more
europepmc   +1 more source

04188 Hereditary coproporphyria with cerebral aneurysms: successful diagnosis and treatment with givosiran

Poster 16
Aurora del Mar Pérez Ys, Annalisa Crisetti, Maria Grazia Savino, Maria Nardella, Francesco Aucella, Claudio Carmine Guida, Filippo Aucella   +6 more
openaire   +1 more source

2020 FDA TIDES (Peptides and Oligonucleotides) Harvest [PDF]

Al Musaimi O, Al Shaer D, Albericio F, de la Toree BG   +3 more
core  

Efficacy and safety of givosiran for acute hepatic porphyria: Final results of the randomized phase III ENVISION trial [PDF]

Journal of Hepatology, 2023
Background & Aims: Acute hepatic porphyria (AHP) is caused by defects in hepatic heme biosynthesis, leading to disabling acute neurovisceral attacks and chronic symptoms.
David Kuter, Herbert L Bonkovsky, Gayle Ross   +2 more
exaly   +6 more sources

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Givosiran for Acute Intermittent Porphyria

New England Journal of Medicine, 2020
Maria Vanessa Perez-Gomez, Alberto Ortiz
exaly   +3 more sources