FDA Approval Genetics Genotype Life Science 
XTalks
MARCH 3, 2021
The US Food and Drug Administration (FDA) has granted approval to Nulibry (fosdenopterin) for reducing the risk of death associated with molybdenum cofactor deficiency type A (MoCD Type A), making it the first treatment for the ultra-rare, genetic metabolic disorder. The approval was granted to BridgeBio Pharma, Inc.
XTalks
DECEMBER 12, 2023
Importantly, FDA-approved products are now available for these PET imaging applications. The approvals have unlocked the potential to use these highly sensitive agents to assess patient eligibility, determine target engagement and evaluate treatment efficacy for Alzheimer’s disease.
XTalks
APRIL 24, 2023
Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies.
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