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Recent items include:
- Sarepta: A Prove-It Story Now Defined By ELEVIDYS Execution
- Scholar Rock jumps 12% after FDA approves isembyld for spinal muscular atrophy
- Ractigen Therapeutics Presents Positive First-in-Human Data for RAG-18 at WMS 2026, Establishing Clinical Proof-of-Mecha
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Scholar Rock jumps 12% after FDA approves isembyld for spinal muscular atrophy
Ractigen Therapeutics Presents Positive First-in-Human Data for RAG-18 at WMS 2026, Establishing Clinical Proof-of-Mecha...
First clinical evidence demonstrating that a systemically delivered small activating RNA (saRNA) can safely upregulate an endogenous target protein in human skeletal muscle in a mo...
Scholar Rock SMA drug approved by FDA
Projected by some analysts as a future blockbuster, Isembyld is the first “muscle-targeted” treatment for the disease and, in clinical testing, helped improve motor function in peo...
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