Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study

December 23rd, 2020|Categories: Clinical Development, immunostimulation, Pre-Clinical Research, Toxicology|

Adams D, Polydefkis M, GonzĂ¡lez-Duarte A, Wixner J, Kristen AV, Schmidt HH, Berk JL, Losada LĂ³pez IA, Dispenzieri A, Quan D, ConceiĂ§Ă£o IM, Slama MS, Gillmore JD, Kyriakides T, Ajroud-Driss S, Waddington-Cruz M, Mezei MM, PlantĂ©-Bordeneuve V, Attarian S, Mauricio E, Brannagan TH 3rd, Ueda ...

QR-313, an antisense oligonucleotide, shows therapeutic efficacy for treatment of dominant and recessive dystrophic epidermolysis bullosa: a preclinical study

October 13th, 2020|Categories: Alternative splicing, Pre-Clinical Research, Splice modulation|

Bornert O, Hogervorst M, Nauroy P, Bischof J, Swildens J, Athanasiou I, Tufa SF, Keene DR, Kiritsi D, Hainzl S, Murauer EM, Marinkovich MP, Platenburg G, Hausser I, Wally V, Ritsema T, Koller U, Haisma EM, Nyström A. J Invest Dermatol. 2020 Sep 15:S0022-202X(20)32061-3. Pubmed ...

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