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In 2016, scientists behind a study called the Resilience Project analysed genetic data from 589,000+ people and found 13 adults who carried genetic variants that should have resulted in serious – even deadly – childhood disease, but who were apparently healthy. Giving participants something in return.
Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare geneticdiseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with geneticdiseases.
BridgeBio is dedicated to developing therapies for geneticdiseases with unmet needs. In the analysis involving 31 patients, 13 were treated with Nulibry and compared to 18 genotype-matched untreated patients from the history control group. The approval was granted to BridgeBio Pharma, Inc.
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