The specificity of antisense approaches is much debated and has hampered their development for clinical therapeutics. In the August 14 Proceedings of the National Academy of Sciences, Yee Cho and ...
The antisense molecule tofersen was tested intrathecally in 108 patients with ALS due to SOD1 mutations. In a subgroup with faster-progressing disease, there was no clinical difference from placebo ...
Researchers from Splisense Ltd. and affiliated organizations recently reported preclinical data for SPL-84, an inhaled antisense oligonucleotide drug candidate being developed for the treatment of ...
AMX0114 is an investigational antisense oligonucleotide targeting calpain-2, a calcium-activated protease believed to play a role in ALS disease progression. The Food and Drug Administration (FDA) has ...
Researchers from Osaka University have developed a novel approach to target nicotinamide N-methyltransferase (NNMT), an enzyme implicated in cancer progression, using antisense oligonucleotides (ASOs) ...
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