An antisense therapy restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome. This breakthrough was possible because of the novel findings, also presented ...

When the drug fomivirsen was approved by the FDA in 1998 for the treatment of cytomegalovirus retinitis in patients with HIV/AIDs, it was hailed as a milestone in drug discovery because it was the ...

Patients treated with high-dose BIIB080 saw positive trends on the global Clinical Dementia Rating Sum of Boxes, Mini-Mental State Exam cognitive scales and Functional Activities Questionnaire at week ...

Patients receiving a kidney transplant and who are positive for BK virus (BKV) are at risk of losing their transplant due to BKV reactivation. At this time, there are no antiviral options available ...

Hoth Therapeutics Inc. (NASDAQ:HOTH) on Tuesday announced combined findings from multiple preclinical programs evaluating its precision antisense candidate HT-KIT. HT-KIT is a precision antisense ...

Courtesy of Antisense Therapeutics Ltd. Antisense strategies use polymeric nucleic acids or nucleic acid analogs to bind to and silence specific messenger RNAs. The silencing can be caused either by ...

Targeting a non-encoding stretch of RNA may help shrink tumors caused by an aggressive type of brain cancer in children, according to new research in mice reported March 8 in Cell Reports by Johns ...

HT-KIT triggered significant tumor cell death in preclinical models of gastrointestinal stromal tumors (GIST) and systemic mastocytosis as early as 24 hours post-treatment, with statistically ...