Investigational new drugs for the treatment of Dravet syndrome : an update
INTRODUCTION: While there are already approved anticonvulsants for treatment of children with Dravet syndrome, disease modifying therapy is at its beginning.
AREAS COVERED: This narrative review is updating the latest information about efficacy and safety of both anticonvulsant and disease modifying investigational drugs for Dravet syndrome. Relevant publications were searched for in MEDLINE, GOOGLE SCHOLAR, SCINDEKS, and CLINICALTRIALS.GOV databases, from the dates of their foundation till January 2023.
EXPERT OPINION: The main advancements were made in the treatment of Dravet syndrome with confirmed haploinsufficiency of SCN1A gene. The application of antisense oligonucleotides has so far proven to be the most successful within disease-modifying therapy, but it also requires further refinement of the methodology of application and delivery to target cells, as well as additional testing of the effectiveness of antisense oligonucleotides outside of TANGO technology. Also, the full potential of gene therapy has yet to be explored, given that high capacity adenoviral vectors that can incorporate the SCN1A gene have recently been prepared.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2023 |
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| Erschienen: |
2023 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:32 |
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| Enthalten in: |
Expert opinion on investigational drugs - 32(2023), 4 vom: 15. Apr., Seite 325-331 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Janković, Slobodan M [VerfasserIn] |
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| Links: |
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| Themen: |
Anticonvulsants |
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| Anmerkungen: |
Date Completed 14.04.2023 Date Revised 14.04.2023 published: Print-Electronic Citation Status MEDLINE |
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| doi: |
10.1080/13543784.2023.2193680 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM354381342 |
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| 500 | |a Date Revised 14.04.2023 | ||
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| 500 | |a Citation Status MEDLINE | ||
| 520 | |a INTRODUCTION: While there are already approved anticonvulsants for treatment of children with Dravet syndrome, disease modifying therapy is at its beginning | ||
| 520 | |a AREAS COVERED: This narrative review is updating the latest information about efficacy and safety of both anticonvulsant and disease modifying investigational drugs for Dravet syndrome. Relevant publications were searched for in MEDLINE, GOOGLE SCHOLAR, SCINDEKS, and CLINICALTRIALS.GOV databases, from the dates of their foundation till January 2023 | ||
| 520 | |a EXPERT OPINION: The main advancements were made in the treatment of Dravet syndrome with confirmed haploinsufficiency of SCN1A gene. The application of antisense oligonucleotides has so far proven to be the most successful within disease-modifying therapy, but it also requires further refinement of the methodology of application and delivery to target cells, as well as additional testing of the effectiveness of antisense oligonucleotides outside of TANGO technology. Also, the full potential of gene therapy has yet to be explored, given that high capacity adenoviral vectors that can incorporate the SCN1A gene have recently been prepared | ||
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