UMDF Mitochondrial Medicine 2024 - Therapeutic Development for Mitochondrial Disease: Lessons learned from other Rare Diseases - Course 7
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Therapeutic Landscape for Mitochondrial Disease (8:34)
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Recounting the Adventure of SMA from a Deadly to Manageable Disease (45:42)
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Efforts Toward Approved Therapies for Barth Syndrome (23:35)
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The Drug Approval Journey in Friedreich's Ataxia and New Insights into Post-Approval Challenges (23:24)
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Lessons Learned from the Drug Approval Journey in Metabolic Disorder (28:05)
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Therapeutic Development Panel Discussion (61:25)
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Required Information for CME
