Clinical Trials Genetic Disease Genotype Life Science 
XTalks
APRIL 24, 2023
Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.
XTalks
MARCH 3, 2021
BridgeBio is dedicated to developing therapies for genetic diseases with unmet needs. The efficacy of Nulibry in the treatment of MoCD Type A was demonstrated in data from three different clinical trials that was compared to data from a natural history study. The approval was granted to BridgeBio Pharma, Inc.
XTalks
APRIL 15, 2021
million people in the US, yet there is no treatment for the most common form of the disease: heart failure with preserved ejection fraction (HFpEF). A lack of efficacy is the greatest reason for clinical trial failures, especially in Phase II and Phase III,” says Dr. Tyl. Heart failure affects about 6.2
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