Two molecular control factors play a decisive role in what is known as splicing, the cutting and assembly of mature messenger ...

They succeeded once they were able to isolate the splicing complexes of the fungus that were in the midst of quality control ...

Wave Life Sciences’ Phase II open-label trial investigating its disease-modifying drug for boys living with Duchenne Muscular ...

Amid readouts from the Muscular Dystrophy Association’s annual meeting, the Clinical Trials Arena evaluates five Duchenne trials in 2025.

Avidity Biosciences, Inc.'s innovative AOC platform and promising clinical programs make it a high-risk, high-reward ...

The development of ocular signs of neurofibromatosis type 1 (NF1) is only significant in patients 7 years of age and younger.