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Meeting ID: 221 747 865 238 6 
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Pediatrics
Radhika Dhamija, M.B.B.S. & Margot A. Cousin, Ph.D.
"From ASO Therapy to Trial Readiness in ADLD: A Translational Framework for Rare Diseases"

Learning objectives: 
Upon conclusion of this activity, participants will be able to:

• Describe the path of individualized ASO therapy development to a single-patient trial in LMNB1-related ADLD.
• List components of rare disease translational research.
• Translate the ADLD framework to other rare neurological diseases.

Radhika Dhamija, M.B.B.S. & Margot A. Cousin, Ph.D. do not have any relevant financial relationships to disclose and do not intend to discuss any off-label/investigative use of Pharmaceuticals or Instruments. 

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TRANSCRIPT
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