Hunter Syndrome

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Curran Simpson, Regenxbio's president and CEO

Dismay as FDA blocks Regenxbio's Hunter syndrome therapy

The FDA said it needs more information before it can approve Regenxbio's gene therapy for Hunter syndrome, in a devastating decision for patients.

R&D

This week in clinical trials: 5th Feb to 9th Feb 2024

In this clinical trial round-up, we look at life sciences research and development announced during the week of 5th to 9th February, from early-stage onwards.

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Shire's Hunter Syndrome cognitive impairment drug fails

Trial missed primary and secondary endpoints

Dismay as FDA blocks Regenxbio's Hunter syndrome therapy

This week in clinical trials: 5th Feb to 9th Feb 2024

Shire's Hunter Syndrome cognitive impairment drug fails

NHS "isn't delivering equitable care for rare diseases"

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