RNA technology is regarded as one of the newest frontiers in medicine, but in fact a primordial innovator got there way ...

PTC Therapeutics, Inc. today reported positive topline results from the 24-month interim analysis of the PIVOT-HD long-term extension study, with favorable dose-dependent effects on disease ...

Researchers discover that the protein Adgrl2 uses alternative splicing to switch between building brain synapses and sealing ...

PTC Therapeutics, Inc. will host a webcast conference call today, April 28 at 4:30 p.m. ET to share results from the 24-month interim analysis of the PIVOT-HD long-term extension study of votoplam.

SEED Therapeutics, Inc. (“SEED”), a clinical-stage biotechnology company pioneering rationally designed molecular glue ...

A study identifies PCBP1 as a regulator of AARS2 function in heart cells, linking its loss to mitochondrial damage, and ...

An isogenic iPSC and AI-enabled disease modeling platform spanning multiple genetically defined CMT subtypes will be ...

In this valuable study, the authors develop new approaches to investigate mRNA imprinting, a phenomenon in which RNA-protein complexes form in the nucleus to influence the fate of transcripts in the ...

Researchers developed STRIPE, a targeted long-read RNA sequencing tool that identifies disease-causing variants missed by ...

AI-generated content from the United States and Iran is shaping public perception of the conflict and overshadowing reporting ...

Not revised: This Reviewed Preprint includes the authors’ original preprint (without revision), an eLife assessment, and public reviews. In this manuscript, the authors aimed to show that SF1 and QKI ...