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Peds
Marc Patterson, M.D.
"Developing Therapy for Ultra-Rare Inherited Metabolic Disease: Lessons from Niemann-Pick Disease, Type C"

Learning objectives:

• Recognize the challenges in designing and executing clinical trials in ultra-rare diseases
• Describe the pathophysiology of Niemann-Pick disease type C
• Review the strengths and weaknesses of clinical trials in Niemann-Pick disease type C, and extrapolate this experience to ultra-rare diseases more generally

Dr. Patterson (and/or spouse/partner) has the following relevant financial relationships to disclose: Consultant - IntraBio, Orphazyme, Recursion; Grant/Research Support - Amicus, Glyocmine, Idorsia, Orphazyme, Shire-Takeda; Stock Shareholder - IntraBio; Other - Sage: Editor in Chief, Journal of Child Neurology and Child Neurology Open; Wiley/SSIEM; Editor, Journal of Inherited Metabolic Disease and JIMD Reports; UpToDate.

He does intend to discuss off-label/investigative uses of the following commercial products/devices: Actelion - Miglustat; Mallinckrodt - beta hydroxypropyl cyclodextrin; CycloTherapeutics - beta hydroxypropyl cyclodextrin; IntraBio - N-acetyl-L-Leucine; Orphazyme - Arimoclomol; Merck - Vorinostat; Generic food supplements; multiple manufacturers - N-acetylcysteine.

ATTENDANCE / CREDIT
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TRANSCRIPT
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