Alternative splicing (AS) of pre-mRNA plays a crucial role in tissue-specific gene regulation, with disease implications due to splicing defects. Predicting and manipulating AS can therefore uncover ...

At this week’s Muscular Dystrophy Association Clinical and Scientific Conference in Dallas, researchers from Suzhou Genassist Therapeutics Co. Ltd. presented preclinical data for GEN-6050X ...

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

The company plans to submit a biologics licence application to the US Food and Drug Administration later this year.

Dyne is eyeing an accelerated approval filing for DYNE-251 in early 2026 that would pit the asset against Sarepta’s Exondys ...

The Phase I/II trial sought to establish the impact of DYNE-251 on mobility-related endpoints, seeing an increase over ...

Avidity Biosciences (RNA) announced del-zota topline data from the Phase 1/2 EXPLORE44 trial in people living with Duchenne muscular dystrophy ...

Avidity Biosciences (RNA) announced positive del-zota topline data from the Phase 1/2 EXPLORE44 trial in people living with Duchenne muscular ...

Avidity Biosciences shared Monday that its RNA drug for people with certain mutations of Duchenne muscular dystrophy ...

While data on functional endpoints are still to come, Avidity Biosciences Inc. executives said the firm is moving ahead with ...

Learn more about whether PTC Therapeutics, Inc. or Sarepta Therapeutics, Inc. is a better investment based on AAII's A+ ...

Dyne Therapeutics’ DYNE-101 shows positive results in phase 1/2 ACHIEVE study. Read why DYN stock may rise with FDA approval and a $2.78B market opportunity.