BioMarin and n-Lorem partner to develop ASO treatment for ReNU syndrome

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

July 27, 2026 9:05 AM EDT

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 cause of this serious neurodevelopmental condition

SAN RAFAEL, Calif. and SAN DIEGO, July 27, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq:... More