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Press Releases

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  • Topic: Treatments
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31 Mar, 2025

National MPS Society Responds to Dr. Peter Marks’ Resignation

The Society praises Dr. Marks' leadership at the FDA and urges continued strong leadership to advance MPS...

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7 Nov, 2024

Researcher and Advocate: Dr. Kakkis

In the early days of his career, a young scientist named Emil Kakkis found himself deep in the world of...

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5 Sep, 2024

Update on RGX-121 Treatment for MPS II

The National MPS Society is pleased to share an update on RGX-121, an investigational gene therapy for MPS II...

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4 Sep, 2024

Denali Therapeutics Announces Plans to File for Accelerated Approval of Tividenofusp Alfa (DNL310) for the Treatment of MPS II

After recent successful meetings with the FDA, Denali Therapeutics announced its plan to file for accelerated...

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24 Jul, 2024

Update on Allievex and AX 250

 An Update on Allievex  Dearest Sanfilippo Community Members and Allies, It is with deep...

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