The US Food and Drug Administration (FDA) has approved biotech BioMarin’s Palynziq (pegvaliase-pqpz) for adults with the rare and serious genetic disease phenylketonuria (PKU). Patients with PKU ...
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
NGGT has initiated Phase I/II clinical trials in the U.S. and China to evaluate the safety and efficacy of NGGT002 for the <a ta ...
About Phenylketonuria Phenylketonuria (PKU) is a rare, inherited metabolic disease, which affects the ... that provide benefits to children and adults living with rare disorders.
The US Food and Drug Administration (FDA) has approved biotech BioMarin’s Palynziq (pegvaliase-pqpz) for adults with the rare and serious genetic disease phenylketonuria (PKU). Patients with PKU ...
About Phenylketonuria Phenylketonuria (PKU) is a rare, inherited metabolic disease, which affects the brain ... differentiated medicines that provide benefits to children and adults living with rare ...
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