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- Following treatment, the N-acetylaspartate levels of CANaspire participants are consistent with levels seen in individuals with milder Canavan disease based on findings from the Company’s... | May 22, 2023
The groundbreaking treatment is one of six gene therapies cleared since 2017 that have transformed the outlook for disorders once considered hopeless — and also renewed questions about how the health care system can afford such breathtakingly expensive medicines.
Guangping Gao, PhD, will be honored by the National Tay-Sachs & Allied Diseases Association for his work in identifying the Canavan gene, revitalizing gene therapy, and his many accomplishments leading to potential treatments for Canavan disease.
POXEL SA (Euronext POXEL FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis
/PRNewswire/ -- BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced...