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5min chapter
Peter Mantas - The Potential Opportunity in Life Sciences
The Business Brew
Backlog of Drug Approvals and the Impact on Rare Disease Treatments
This chapter discusses the backlog of drug approvals in phase three and the impact on rare disease treatments. It highlights the case of Clearpoint Narrow, a gene therapy drug for a rare genetic disease, which has been approved in Europe and Israel but not yet by the FDA in the United States. The chapter also addresses the high costs of rare disease drugs, insurance coverage challenges, and the financial burden faced by families seeking treatment.
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