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Protesters Demand Faster FDA Approval for Rare Disease Therapies

3/19/2026

24 4 Full Breakdown

Summary

Story summary

Advocates marched to the U.S. Food and Drug Administration (FDA) to protest delays in approving RGX-121 for Hunter Syndrome and therapies for mucopolysaccharidoses (MPS).
They demand regulatory flexibility and congressional oversight.
The FDA defends its decisions, citing the need for strong clinical evidence.
Families feel blindsided by recent rejections and fear they may lose access to therapies.

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Story summary

Advocates marched to the U.S. Food and Drug Administration (FDA) to protest delays in approving RGX-121 for Hunter Syndrome and therapies for mucopolysaccharidoses (MPS).
They demand regulatory flexibility and congressional oversight.
The FDA defends its decisions, citing the need for strong clinical evidence.
Families feel blindsided by recent rejections and fear they may lose access to therapies.