It is estimated that over 11,000 children across Europe, Middle East, and Africa are affected by achondroplasia and could be eligible for treatment with Voxzogo
Seen in 1 in 3 million births, the malformation was detected at the antenatal stage during the mother's pregnancy
Idiopathic hypersomnia is a life-long condition, and the approval of Xywav will be instrumental in providing treatment for symptoms such as excessive sleepiness and difficulty waking, and in effectively managing this debilitating disorder
Partnership enables broad reach of cfDNA methylation blood tests for early cancer detection
Nexviazyme specifically targets the M6P receptor, the key pathway for enzyme replacement therapy, to effectively clear glycogen build-up in muscle cells
Advances deployment of mRNA technology across vaccines and therapeutics development
The facility is expected to be completed in 2024
Sapropterin dihydrochloride powder for oral solution is available in 100 mg unit dose
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