Matches in SemOpenAlex for { <https://semopenalex.org/work/W4323355310> ?p ?o ?g. }
Showing items 1 to 98 of
98
with 100 items per page.
- W4323355310 endingPage "325" @default.
- W4323355310 startingPage "315" @default.
- W4323355310 abstract "Antisense oligonucleotide (ASO) mediated exon skipping aims to reframe dystrophin transcripts for patients with Duchenne muscular dystrophy (DMD). Currently 4 ASOs have been approved by the Food and Drug Administration targeting exon 45, 51 and 53 based on low level dystrophin restoration. Additional studies to confirm functional effects are ongoing. Furthermore, efforts are ongoing to increase muscle specific delivery of ASOs. Consequently, there are 5 clinical trials ongoing or planned for exon 51 skipping ASOs in Europe. While exon 51 skipping applies to the largest group of patients, DMD expert centers do not have sufficient numbers of patients or capacity to run all these trials in parallel. Even at a national level numbers may be too scarce. At the same time, some families now face the choice between participation in different clinical trials of exon 51 skipping, sometimes in addition to the choice of participating in a micro-dystrophin gene therapy trial. In this opinion paper, we outline the challenges, compare the different exon 51 skipping trials, and outline how different European centers and countries try to cope with running multiple trials in parallel for a small group of eligible patients." @default.
- W4323355310 created "2023-03-08" @default.
- W4323355310 creator A5005592998 @default.
- W4323355310 creator A5006695139 @default.
- W4323355310 creator A5014186653 @default.
- W4323355310 creator A5030692760 @default.
- W4323355310 creator A5033643112 @default.
- W4323355310 creator A5040391974 @default.
- W4323355310 creator A5042269980 @default.
- W4323355310 creator A5046698487 @default.
- W4323355310 creator A5054679892 @default.
- W4323355310 creator A5089089477 @default.
- W4323355310 date "2023-05-02" @default.
- W4323355310 modified "2023-10-14" @default.
- W4323355310 title "The Dilemma of Choice for Duchenne Patients Eligible for Exon 51 Skipping The European Experience" @default.
- W4323355310 cites W1997490017 @default.
- W4323355310 cites W2018862827 @default.
- W4323355310 cites W2076827123 @default.
- W4323355310 cites W2118947165 @default.
- W4323355310 cites W2158041351 @default.
- W4323355310 cites W2159741632 @default.
- W4323355310 cites W2322780762 @default.
- W4323355310 cites W2511893227 @default.
- W4323355310 cites W2587479628 @default.
- W4323355310 cites W2725706857 @default.
- W4323355310 cites W2905428362 @default.
- W4323355310 cites W2951224510 @default.
- W4323355310 cites W3004568198 @default.
- W4323355310 cites W3118099865 @default.
- W4323355310 cites W3131782536 @default.
- W4323355310 cites W3155362558 @default.
- W4323355310 cites W3213032771 @default.
- W4323355310 cites W4292342217 @default.
- W4323355310 cites W4292458118 @default.
- W4323355310 cites W4308577337 @default.
- W4323355310 doi "https://doi.org/10.3233/jnd-221648" @default.
- W4323355310 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/36911945" @default.
- W4323355310 hasPublicationYear "2023" @default.
- W4323355310 type Work @default.
- W4323355310 citedByCount "0" @default.
- W4323355310 crossrefType "journal-article" @default.
- W4323355310 hasAuthorship W4323355310A5005592998 @default.
- W4323355310 hasAuthorship W4323355310A5006695139 @default.
- W4323355310 hasAuthorship W4323355310A5014186653 @default.
- W4323355310 hasAuthorship W4323355310A5030692760 @default.
- W4323355310 hasAuthorship W4323355310A5033643112 @default.
- W4323355310 hasAuthorship W4323355310A5040391974 @default.
- W4323355310 hasAuthorship W4323355310A5042269980 @default.
- W4323355310 hasAuthorship W4323355310A5046698487 @default.
- W4323355310 hasAuthorship W4323355310A5054679892 @default.
- W4323355310 hasAuthorship W4323355310A5089089477 @default.
- W4323355310 hasBestOaLocation W43233553101 @default.
- W4323355310 hasConcept C104317684 @default.
- W4323355310 hasConcept C126322002 @default.
- W4323355310 hasConcept C194583182 @default.
- W4323355310 hasConcept C2778776201 @default.
- W4323355310 hasConcept C2778943923 @default.
- W4323355310 hasConcept C2780394045 @default.
- W4323355310 hasConcept C36823959 @default.
- W4323355310 hasConcept C535046627 @default.
- W4323355310 hasConcept C54355233 @default.
- W4323355310 hasConcept C60644358 @default.
- W4323355310 hasConcept C71924100 @default.
- W4323355310 hasConcept C86803240 @default.
- W4323355310 hasConceptScore W4323355310C104317684 @default.
- W4323355310 hasConceptScore W4323355310C126322002 @default.
- W4323355310 hasConceptScore W4323355310C194583182 @default.
- W4323355310 hasConceptScore W4323355310C2778776201 @default.
- W4323355310 hasConceptScore W4323355310C2778943923 @default.
- W4323355310 hasConceptScore W4323355310C2780394045 @default.
- W4323355310 hasConceptScore W4323355310C36823959 @default.
- W4323355310 hasConceptScore W4323355310C535046627 @default.
- W4323355310 hasConceptScore W4323355310C54355233 @default.
- W4323355310 hasConceptScore W4323355310C60644358 @default.
- W4323355310 hasConceptScore W4323355310C71924100 @default.
- W4323355310 hasConceptScore W4323355310C86803240 @default.
- W4323355310 hasIssue "3" @default.
- W4323355310 hasLocation W43233553101 @default.
- W4323355310 hasLocation W43233553102 @default.
- W4323355310 hasLocation W43233553103 @default.
- W4323355310 hasOpenAccess W4323355310 @default.
- W4323355310 hasPrimaryLocation W43233553101 @default.
- W4323355310 hasRelatedWork W2018499417 @default.
- W4323355310 hasRelatedWork W2041137184 @default.
- W4323355310 hasRelatedWork W2045245107 @default.
- W4323355310 hasRelatedWork W2135265213 @default.
- W4323355310 hasRelatedWork W2168692744 @default.
- W4323355310 hasRelatedWork W2494748207 @default.
- W4323355310 hasRelatedWork W2563648084 @default.
- W4323355310 hasRelatedWork W2809419175 @default.
- W4323355310 hasRelatedWork W2904491638 @default.
- W4323355310 hasRelatedWork W3214807650 @default.
- W4323355310 hasVolume "10" @default.
- W4323355310 isParatext "false" @default.
- W4323355310 isRetracted "false" @default.
- W4323355310 workType "article" @default.