Vanda Pharmaceuticals Inc.’s VGT-1849A, a selective antisense oligonucleotide (ASO)-based JAK2 inhibitor, has been awarded ...

As the field of oligonucleotide therapeutics continues to evolve, the integration of robust analytical tools will be ...

The FDA approved olezarsen (Tryngolza) as the first drug for adults with familial chylomicronemia syndrome, a rare genetic ...

The US FDA has approved olezarsen (Tryngolza) as the first treatment for adults with familial chylomicronemia syndrome, a ...

Company on track to initiate the Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and in other age ...

The Food and Drug Administration (FDA) has approved Tryngolza™ (olezarsen) as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).

A novel study from Korea uncovers the process of synthesis and pivotal role of the transfer RNA-derived fragment, ...

PANTHERx® Rare, a leader in rare disease product patient access and support services in the United States, is pleased to ...