[EN] COMPOUNDS AND METHODS FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY<br/>[FR] COMPOSÉS ET PROCÉDÉS POUR LE TRAITEMENT DE LA DYSTROPHIE MUSCULAIRE DE DUCHENNE
申请人:DTX PHARMA INC
公开号:WO2021108640A1
公开(公告)日:2021-06-03
Disclosed herein are compounds including a single-stranded oligonucleotide (A) having a nucleobase sequence complementary to a portion of the dystrophin pre-mRNA, their preparation, and uses thereof for the treatment of Duchenne muscular dystrophy.
Abstract In the present work we investigated the conformations of some N -mono-substituted propanamides of general formula CH 3 CH 2 CONHR, wherein R is chosen from n -(C 1 C 9 )alkyl, cyclo(C 3 C 6 )alkyl, some branched (C 3 C 6 )alkyl or phenyl. The amides were synthesised by the well known Schotten-Baumann reaction—acylation of the corresponding amines with propionyl chloride. On the basis of
A Comparison of Immobilised Triphenylphosphine and 1‐Hydroxybenzotriazole as Mediators of Catch‐and‐Release Acylation Under Flow Conditions
作者:Joseph Tadros、Christian Dankers、Ashley Jurisinec、Maria Menti‐Platten、Janice R. Aldrich‐Wright、Christopher P. Gordon
DOI:10.1002/asia.202101308
日期:2022.3
A ‘catch-and-release’ assessment of immobilised triphenylphosphine (PS-PPh3) and 1-hydroxybenzotriazole (PS-HOBt) as facilitators of acylation-mediated amide couplings is described. Through evaluations of reaction temperatures, substrate stoichiometries, solvents, and flow rates, we report findings in favour of PS-HOBt over PS-PPh3.
Behaviour of the extractant Me-TODGA upon gamma irradiation: quantification of degradation compounds and individual influences on complexation and extraction
Diglycolamides (DGAs), and in particular N,N,N′,N′-tetraoctyl diglycolamide (TODGA), are well-known candidates for the co-extraction of trivalent actinides (An(III)) and lanthanides (Ln(III)) from highly acidic aqueous solutions of nuclear waste. A derivative of TODGA, the so-called Me-TODGA with the addition of a methyl-substituent on the central part of the TODGA molecule, has been proposed to improve
Gamma-radiolytic stability of new methylated TODGA derivatives for minor actinide recycling
作者:Hitos Galán、Christopher A. Zarzana、Andreas Wilden、Ana Núñez、Holger Schmidt、Richard J. M. Egberink、Andrea Leoncini、Joaquín Cobos、Willem Verboom、Giuseppe Modolo、Gary S. Groenewold、Bruce J. Mincher
DOI:10.1039/c5dt02484f
日期:——
Open the door to the understanding of radiolytic ruptures of extracting ligands under gamma radiation.