Imvax has secured $29m in a financing round from current investors, aimed at advancing the Phase IIb trial of its autologous ...

Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...

Discover the importance of vision, genetic eye disorders, and RNA-based therapies for precision treatment in India and ...

The gene therapy improved motor functions in children with Duchenne muscular dystrophy two years after treatment ...

In trials at University College London Hospitals (UCLH), an ASO is being used to target the mutated gene that results in the ...

Biogen’s applications for the higher dose regimen of SMA drug Spinraza are now under review in the US and Europe.

A drug that halts an AIDS-related eye infection could be the first antisense therapy to reach the market. However, whether the drug can truly be called "antisense" depends on some specifics--such as ...

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Delve into the increasingly dynamic landscape of ALS treatment development with thirteen companies advancing candidates in ...

In this review of RNA therapies, we paint an overview of what’s going on in a field that is addressing a number of diseases.

An investigational higher dose of spinal muscular atrophy drug nusinersen gains attention as the FDA and European Medicines ...

They investigated the effects of antisense oligonucleotides targeting the regulatory RIα subunit of cAMP-dependent protein kinase (PKA). They treated human PC3 prostate cancer cells with antisense ...