BioMarin and n-Lorem Foundation Enter Early Research Collaboration to Develop Potential First-in-Disease Medicine for Newly Identified ReNU Syndrome

ReNU syndrome was discovered in 2024 and there are no medicines approved for the condition Collaboration combines BioMarin's leadership in genetic medicines with n-Lorem's pioneering antisense expertise Investigational antisense oligonucleotide (ASO) aims to address the underlying genetic...

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