Results 91 to 100 of about 1,115 (145)

RNA interference therapy in acute hepatic porphyrias.

Blood, 2023
Yasuda M, Keel S, Balwani M.
europepmc   +1 more source

RNA therapeutic targeting of recalcitrant and rare cancers. [PDF]

Explor Target Antitumor Ther
Ishola AA, Verusingam ND, Lawal B.
europepmc   +1 more source

Givosiran treatment over 36 months for people with acute hepatic porphyria: a plain language summary

Future Rare Diseases
Kristen Wheeden, Sue Burrell, Susana Monroy, Paolo Ventura, Marianne T Sweetser, Gang Jia, Manish Thapar, Nicole Castellano   +7 more
doaj   +1 more source

Hepatic Porphyria Presenting with Persistent Abdominal Pain: A Case Report and Literature Review. [PDF]

Iran J Pathol
Yu Y, Yu L, Li M, Ma M, Zhu Y, Shen J.
europepmc   +1 more source

Separation and Characterization of Therapeutic Oligonucleotide Isomer Impurities by Cyclic Ion Mobility Mass Spectrometry. [PDF]

J Am Soc Mass Spectrom
Omuro S, Yamaguchi T, Kawase T, Hirose K, Yoshida T, Inoue T, Obika S.   +6 more
europepmc   +1 more source

Translational and clinical development of therapeutic siRNA and ASOs: current industry practices, perspectives, and recommendations. [PDF]

Nucleic Acids Res
Christensen JK, Colletti N, Hooshfar S, Jiang RR, Kuo C, Lindmark B, Lumen A, Youssef AS, Albertolle M, Aluri KC, Chen L, Chen J, Chopda G, Chua XY, Morais P, Dubois B, Gibbons FF, Gudey SK, Gupta S, Hood S, Humphreys S, Husser C, Huth F, Jani D, Jian W, Kakuda T, Kurz J, Linnebjerg H, Liu J, Lohmann S, Niu T, Root K, Singla S, Tang C, Veluri R, Zhang GS, Zhang S, Jawa V.   +37 more
europepmc   +1 more source

04103 Effect of Givosiran for hereditary coproporphyria: 2 cases report

Poster 9
Tomohide Adachi, Nobuaki Ozaki
openaire   +1 more source

A New Generation of Porphyrias: A Case of Acute Intermittent Porphyria. [PDF]

Cureus
Sousa M, Ribeiro F, Pais T, Romão S, Oliveira A.   +4 more
europepmc   +1 more source

Pathogenesis and clinical management of liver damage in porphyrias: Mechanisms and therapeutic approaches. [PDF]

World J Hepatol
Zeng T, Huang SY, Chen JN, Pang JH, Chong YT, Li XH.   +5 more
europepmc   +1 more source

Systemic messenger RNA replacement therapy is effective in a novel clinically relevant model of acute intermittent porphyria developed in non-human primates. [PDF]

Gut
Córdoba KM, Jericó D, Jiang L, Collantes M, Alegre M, García-Ruiz L, Manzanilla O, Sampedro A, Herranz JM, Insausti I, Martinez de la Cuesta A, Urigo F, Alcaide P, Morán M, Martín MA, Lanciego JL, Lefebvre T, Gouya L, Quincoces G, Unzu C, Hervas-Stubbs S, Falcón-Pérez JM, Alegre E, Aldaz A, Fernández-Seara MA, Peñuelas I, Berraondo P, Martini PGV, Avila MA, Fontanellas A.   +29 more
europepmc   +1 more source