BioMarin, n-Lorem Partner to Develop First Investigational Therapy for ReNU Syndrome
BioMarin and the n-Lorem Foundation have partnered to develop an investigational antisense oligonucleotide therapy targeting the most common genetic variant associated with ReNU syndrome, a rare neurodevelopmental disorder with no approved treatments.
BioMarin Pharmaceutical | 28/07/2026 | By News Bureau
US FDA Accepts BioMarin's sNDA for VOXZOGO in Achondroplasia
The US Food and Drug Administration (FDA) has accepted BioMarin's sNDA for VOXZOGO in achondroplasia, supported by long-term safety and efficacy data, including adult height, body proportionality, and arm span outcomes.
BioMarin Pharmaceutical | 14/07/2026 | By News Bureau
BioMarin Strengthens its Leadership with New Appointments
At BioMarin, he will assume responsibility for the company's R&D organization, including discovery research, preclinical, translational and clinical programs, as well as global regulatory and medical affairs.
BioMarin Pharmaceutical | 22/08/2024 | By Aishwarya | 833
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