Results 101 to 110 of about 1,551 (185)

Givosiran per al tractament de la porfíria hepàtica aguda

, 2022
Les porfíries són un grup de malalties metabòliques causades per una pèrdua de funcionalitat en els enzims que participen en la biosíntesi del grup hemo. Es classifiquen en funció de les principals manifestacions clíniques (neurològiques o cutànies) i del teixit on es produeix l’alteració enzimàtica (fetge o medul·la òssia).
openaire   +1 more source

Small interfering RNA: Discovery, pharmacology and clinical development—An introductory review [PDF]

, 2022
Addison, Melisande L., Dear, James W., Ranasinghe, Priyanga, Webb, David J.   +3 more
core   +1 more source

Nucleic Acid Conjugates: Unlocking Therapeutic Potential [PDF]

Nucleic acids have emerged as a powerful class of therapeutics. Through simple base pair complementarity, nucleic acids allow the targeting of a variety of pathologically relevant proteins and RNA molecules.
Booth, Michael J., Kashyap, Disha
core   +2 more sources

Givosiran [PDF]

, 2020
openaire   +1 more source

PB2251: SOMETIMES IT’S ZEBRAS: SUCCESSFUL TREATMENT OF ERYTHROPOIETIC PROTOPORPHYRIA POST PRIMARY MYELOFIBROSIS WITH GIVOSIRAN - A CASE REPORT

HemaSphere, 2023
Alina Catana, Geanina Mera, Cristina Popa   +2 more
doaj   +1 more source

Real-World Experience With Givosiran in Acute Porphyrias: A Narrative Review and a Novel Hypothesis. [PDF]

Cureus
Petrides PE.
europepmc   +1 more source

Psychiatric Symptoms in Acute Intermittent Porphyria - Case Report and Course of Treatment Using Placebo [PDF]

Introduction: Acute intermittent porphyria is a disease inherited in an autosomal dominant manner, occurring with a frequency of 1:75,000 people.
Dominika Prystacka-Szar, Kosiba, Waldemar, Kupczak, Wojciech, Siemko, Jakub   +3 more
core   +1 more source

Claw hands in acute intermittent porphyria. [PDF]

Oxf Med Case Reports
Wang JD, Chou CT.
europepmc   +1 more source

Enhanced liver-targeted delivery with ribofuranose-based GalNAc conjugates. [PDF]

Mol Ther Nucleic Acids
Pradeep SP, Ruchi R, Bahal R.
europepmc   +1 more source

Oligonucleotides: evolution and innovation [PDF]

Oligonucleotides, comprising single or double strands of RNA or DNA, are vital chemical compounds used in various laboratory and clinical applications. They represent a significant class of therapeutics with a rapidly expanding range of uses.
Al Musaimi O
core