Patients in the Phearless study of BMN 307 – an adeno-associated virus (AAV) gene therapy designed to replace the phenylalanine hydroxylase (PAH) deficient in people with PKU – are getting a ...
About 100 university experts and scholars from China and around the world gathered at Peking University PKU on Wednesday advocating for more cooper ...
There are only around 17,000 people living with PKU in the US, and 150,000 globally, but - despite that - treatments can command healthy sales. Meanwhile, the positive results in PKU are a boost ...
In the village of He Ci in Hengnan county, Central China's Hunan Province, a family exemplifies love and wisdom across generations. At the center of this four-generation household is nearly ...
With a growing portfolio in an attractive rare-disease niche—and acceleration of profit growth beginning in 2022—BioMarin could be an acquisition target for pharmaceutical firms with pipelines to fill ...
About 40% of autistic people also experience significant anxiety. This is the case even though anxiety is not a part of the criteria for diagnosing autism—nor is it one of the descriptive options ...