Researchers at Baylor College of Medicine and their collaborators tested a novel approach to restore normal heart function in ...

Ubiquitin marks proteins for degradation, whereby ubiquitin molecules can be combined in different types and numbers forming ...

A new discovery about how tiny protein clusters form in cells could pave the way for treatments for Emery-Dreifuss muscular dystrophy (EDMD), a rare genetic disorder that causes muscle weakness and ...

Activating a protein called potassium channel Kv2.1 may help improve motor function in SMA, making it a potential SMA ...

Researchers at Baylor College of Medicine and their collaborators focused on Myotonic Dystrophy 1 heart problems testing a novel approach to restore normal function.

Researchers at the Max Planck Institute of Biochemistry (MPIB) have developed the new UbiREAD technology to decode the various combinations of ubiquitin molecules – the ubiquitin code – which ...

Use of DYNE-101 is leading to improvements in muscle, and specifically finger, function in myotonic dystrophy type 1 patients ...

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

The firm's stock price dropped 27 percent on Tuesday following the news that the young man died from acute liver failure after getting Elevidys.

RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, per interim data.

Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease----Data Presented at the 2025 Muscular ...