Italfarmaco S.p.A. today announced a comprehensive update on the regulatory and clinical advancements for givinostat, the ...

Protein extracted from dystrophin sufficient skeletal muscle was incubated overnight with terminal ... Although these data are encouraging, the increased efficacy of gene therapy observed by blocking ...

2024 — Researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people with ...

Q4 2024 Earnings Call Transcript March 4, 2025 Wave Life Sciences Ltd. beats earnings expectations. Reported EPS is $0.17, expectations were $-0.17. Operator: Good morning and welcome to the Wave Life ...

Protein folding is the process by which proteins ... in bridging multilevel hierarchical mineralization, but the mechanism orchestrating the homogeneous morphology of mesoscale mineralized motifs ...

Dosing underway in INLIGHT trial of WVE-007 in obesity with clinical data expected in 2H 2025; enrollment complete in first single dose ...

The upcoming 48 week data will include dystrophin from muscle ... the total level was M-protein. That's highly indicative of the mechanism of action of editing, so it's the best way to benchmark ...

Gene silencing is a negative feedback mechanism that regulates gene expression to define cell fate and also regulates metabolism and gene expression throughout the life of an organism. In plants, gene ...

Protein misfolding and formation of cross-β structured amyloid fibrils are linked to many neurodegenerative disorders but are also the building blocks of novel biomaterials. Our research aims to ...

M-AAT protein and potentially extended dose intervals. We remain on track to report our DMD 48-week clinical results of WVE-N531 this month. With both our progress in the clinic and in advancing a ...

Dosing underway in INLIGHT trial of WVE-007 in obesity with clinical data expected in 2H 2025; enrollment complete in first single dose cohort Multi-dosing ongoing in 200 mg cohort of RestorAATion-2 ...