Investors consider Dyne Therapeutics' treatments for muscle diseases and strong cash position as a speculative Buy ...

Crispr has already secured a historic first approval for a CRISPR/Cas9 drug and has several more potential blockbuster ...

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD), as it aims for a launch in the ...

New Gene Therapy Approach Shows Promise for Duchenne Muscular Dystrophy July 24, 2024 — Researchers have made a significant breakthrough in developing a new gene therapy approach that restores ...

Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD) will be an option for many more patients in the US after the FDA expanded the breadth of its approved labelling. Elevidys ...

Belief Biomed Inc.’s gene therapy drug BBM-D101 has been awarded U.S. orphan drug and rare pediatric disease designations by the FDA for the treatment of Duchenne muscular dystrophy (DMD).

We identified a novel and significant association between Duchenne muscular dystrophy and autism spectrum ... a specific region within the dystrophin gene that is altered, creating either a ...

To understand the workings of DNA in relation to disease, scientists at Los Alamos National Laboratory have developed the first multimodal deep learning model of its kind, EPBDxDNABERT-2, capable of ...

A protein called TDP-43 is usually located in the nucleus of motor neurons, where it regulates gene expression required ... CHMP7 was restored to its usual location in the cytoplasm, leaving ...

lakshmiprasad S / Getty Images Asset location is a tax-minimization strategy that helps investors optimize their portfolio by considering the tax treatment of different types of investments.

Gene regulation refers to the mechanisms that act to induce or repress the expression of a gene. These include structural and chemical changes to the genetic material, binding of proteins to ...