Harvard Medical School researchers have uncovered crucial insights into how an emerging class of antiviral drugs works.

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Patients who have a particular genetic disease whose symptoms include progressively worsening muscle control were able to walk faster following treatment with an experimental Ionis Pharmaceuticals ...

Kirstyn Hill has worked as a clinical pediatric and women’s health pharmacist since 2020. She was driven to work with a patient population that is not often able to easily speak up for themselves and ...

The investigational antisense oligonucleotide (ASO) therapy BIIB078 may have failed in amyotrophic lateral sclerosis (ALS) clinical trials because it wasn’t adequately reversing key disease processes ...

Brian W. Stone does not work for, consult, own shares in or receive funding from any company or organization that would benefit from this article, and has disclosed no relevant affiliations beyond ...

How New Innovations Are Making Oligonucleotide Synthesis Faster, Cleaner, and More Scalable Oligonucleotides are short strands of DNA or RNA that scientists use to influence biological processes at ...

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Delivering cutting-edge peer-reviewed research on using nucleic acids and related compounds to alter gene expression for therapeutic purposes. A new article in the peer-reviewed journal Nucleic Acid ...

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 ...