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📄Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments
#n=1 #ASO
📄Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments
#n=1 #ASO
Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments
The N=1 Collaborative has developed the N1C VARIANT guidelines, an international consensus
guideline for assessing pathogenic genetic DNA variants toward antisense oligonucleotide
treatment eligibilit...
www.cell.com
March 25, 2025 at 3:25 PM
Reposted
Congratulations Marlen Lauffer and other co-authors for this publication on the N=1 collaborative Variant Guidelines, which contains a step by step approach to assess if in principle a variant is actionable by an antisense oligonucleotide therapy approach
www.sciencedirect.com/science/arti...
www.sciencedirect.com/science/arti...
Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments
Of the around 7,000 known rare diseases worldwide, disease-modifying treatments are available for fewer than 5%, leaving millions of individuals witho…
www.sciencedirect.com
March 25, 2025 at 3:17 PM
Congratulations Marlen Lauffer and other co-authors for this publication on the N=1 collaborative Variant Guidelines, which contains a step by step approach to assess if in principle a variant is actionable by an antisense oligonucleotide therapy approach
www.sciencedirect.com/science/arti...
www.sciencedirect.com/science/arti...