The increasing incidence of genetic and rare diseases is a major factor driving revenue growth in the Antisense ...

Biogen has pointed to an early readout from a small phase 1 trial as evidence that its antisense oligonucleotide salanersen could help children with spinal muscular atrophy (SMA) who are still ...

Ultragenyx Pharmaceuticals RARE announced that the FDA has granted the Breakthrough Therapy designation to its ...

Seizures initially occurred at a rate of 20 to 25 per hour and declined to five to seven per hour following repeated intrathecal dosing with elsunersen, an antisense oligonucleotide therapy.

BIIB080 is an antisense oligonucleotide therapy designed to target microtubule-associated protein tau mRNA and reduce the production of tau.

The Food and Drug Administration has granted Fast Track designation to AMX0114 for the treatment of amyotrophic lateral sclerosis (ALS).

Cure Rare Disease launched its SCA3 program in 2021 to develop an antisense oligonucleotide (ASO) therapy, with initial funding support provided by Gregory Klassen, a patient living with SCA3.

Panelists discuss how exciting ongoing research efforts are expanding similar gene therapy technologies to other muscular ...

The Bay Area biotech has developed an RNA-based antisense oligonucleotide therapy to restore malfunctioning neurons in ALS patients.

A recent study involving researchers from the University of Basel reveals that slowing down the intracellular transport of ...

BIIB080 is an antisense oligonucleotide therapy designed to target microtubule-associated protein tau mRNA and reduce the production of tau.