Final results from the VALOR trial and its open-label extension demonstrated the clinical benefit of tofersen in adults with ...

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 ...

Researchers from Tokyo Medical and Dental University (TMDU) and Ionis Pharmaceuticals, USA, report a modification wherein replacing the RNA strand of a heteroduplex oligonucleotide with DNA may ...

Antisense oligonucleotide (ASO) therapy has the potential to ameliorate many neurodegenerative diseases at the genetic level to suppress the production of harmful proteins or non-coding RNAs.

WOODBRIDGE, Conn.--(BUSINESS WIRE)--Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the ...

Diffuse intrinsic pontine glioma (DIPG) is a lethal pediatric brain cancer that often kills within a year of diagnosis. DIPG occurs in an area of the brainstem called the pons, which controls many of ...

Study design: classification of genetic variants according to their amenability to splice-switching therapy. Population: 235 individuals with the severe genetic disorder ataxia–telangiectasia.

A Penn Medicine patient with a genetic form of childhood blindness gained vision, which lasted more than a year, after receiving a single injection of an experimental RNA therapy into the eye, ...

Please provide your email address to receive an email when new articles are posted on . Phase 1b trial of BII080 demonstrated dose-dependent and sustained reduction of tau protein in CSF, as well as ...

Researchers have devised a molecular structural modification that boosts the efficacy of antisense oligonucleotide-based drugs by replacing the RNA strand of a heteroduplex oligonucleotide with DNA.