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    • Parents & Caregivers
    • Professionals
    • Pipeline
    • Volunteer
    • Events
    • Contact Us
  • Home
  • Parents & Caregivers
  • Professionals
  • Pipeline
  • Volunteer
  • Events
  • Contact Us

therapeutic strategies

ASO DEVELOPMENT

Most advanced ..status TBC

Coming soon...

DRUG REPURPOSING

Academic  (starting early preclinical )

Link to publication

RNAi Knockdown Replacement

Academic Research

Link to publication

RNAi allele specific silencing

Outreach Ongoing- TBC

Coming soon...

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