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Home » Keywords » MECP2 duplication syndrome

Items Tagged with 'MECP2 duplication syndrome'

ARTICLES

RNA strand
Neurology/Psychiatric

FDA awards orphan drug designation to Huidagene’s CRISPR RNA-editing therapy for MECP2 duplication syndrome

Nov. 2, 2023
The FDA has awarded U.S. orphan drug designation and rare pediatric disease designation to Huidagene Therapeutics Co. Ltd.’s HG-204 (AAV-hfCas13Y-gMECP2), an RNA editing therapy based on CRISPR/Cas13Y, for the treatment of methyl-CpG binding protein 2 (MeCP2) duplication syndrome (MDS).
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Digital brain and silhouette
Neurology/Psychiatric

Ionis to advance four neurology programs into clinic

Oct. 5, 2023
Ionis Pharmaceuticals Inc. has announced its intention to advance four new wholly owned neurology medicines into the clinic by the end of next year.
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Brain-DNA illustration

New model of MECP2 duplication syndrome may advance antisense oligonucleotide treatment

March 31, 2021
The neurodevelopmental disorder MECP2 duplication syndrome (MDS) is caused by duplications spanning the methyl-CpG binding protein 2 gene (MECP2) locus, and researchers have shown that the MDS-like phenotype can be reversed in adult symptomatic mice using MECP2-specific antisense oligonucleotides (MECP2-ASOs).
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