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adicionou um novo artigo Health

2025-11-18 11:06:46 -

Exon-Skipping and Beyond: Tracking the Evolving Standard of Care and Pipeline Dynamics in the Multi-Billion Dollar Duchenne Muscular Dystrophy Market.

The Duchenne Muscular Dystrophy (DMD) standard of care has been fundamentally altered by the introduction of exon-skipping oligonucleotide therapies. These drugs work by coaxing the cell's machinery to "skip" over a specific defective exon in the dystrophin gene, allowing the production of a truncated, but partially functional, dystrophin protein. While only applicable to specific genetic...

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