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Neurology

September 18, 2024

FDA grants orphan drug status to Papillon Therapeutics’ PPL-001

The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Papillon Therapeutics’ PPL-001, aimed at treating Friedreich's ataxia, a rare neurodegenerative disorder.

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PPL-001 is a gene-corrected CD34+ hematopoietic stem and progenitor cell (HSPC) therapy. Credit: PublicDomainPictures from Pixabay.

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FDA grants orphan drug status to Papillon Therapeutics’ PPL-001

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