Molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening genetic disorder that progresses rapidly, results in severe and largely irreversible neurological injury, and has a high infant mortality rate; median overall survival age is about four years

BridgeBio Pharma and Affiliate Origin Biosciences Announce FDA Approval of NULIBRY™ (fosdenopterin), the First and Only Approved Therapy to Reduce...

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