Cold Spring Harbor Laboratory researchers have reported that a bespoke antisense oligonucleotide can disrupt alternative ...

Program to feature presentations from the company management about KRRO-121 for the potential treatment of hyperammonemia, a leading clinical hepatologist about ammonia-driven diseases, and a caregive ...

The global oligonucleotide CDMO market is poised for growth driven by the rising clinical pipeline of RNA-based therapies, ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for myotonic dystrophy type 1 (DM1), the most common adult-onset form of ...

Elevated levels of lipoprotein (a) is a genetic disease that causes cardiovascular disease. Steven Nissen, M.D., addresses why it’s important to find treatments for this genetic risk factor.

Department of Surgery, University of California Los Angeles, Los Angeles, United States The final, formatted version of the article will be published soon. Background/Objectives: Schizophrenia is a ...

The n-Lorem Foundation and the EspeRare Foundation announced today a strategic collaboration to expand access in Europe to ...

Currently available therapies such as niacin and PCSK9 inhibitors such as Repatha (evolocumab) and Praluent (alirocumab) can reduce Lp (a) levels, but their impact is modest. Another challenge in ...

PacBio (NASDAQ: PACB), developer of the world's most advanced sequencing technologies, today announced plans to pursue a strategic collaboration with the n-Lorem Foundation and EspeRare that we ...

Wave expects to initiate a Phase 2a multidose portion of WVE-007 INLIGHT clinical trial in individuals living with obesity with higher BMI and ...

Aperture Therapeutics is advancing APRTX-003, an RNA-based antisense therapy targeting MMP9, as a potential ALS treatment.