and PALYNZIQ, a PEGylated recombinant phenylalanine (Phe) ammonia lyase enzyme delivered through subcutaneous injection to reduce blood Phe concentrations. It also develops BRINEURA, a recombinant ...

There are a couple of drug treatments available, namely BioMarin’s enzyme-based therapies Kuvan (sapropterin) or Palynziq (pegvaliase), which are both approved to reduce levels of phe.

Drug treatment is an option for some patients, with BioMarin's enzyme-based therapies Kuvan (sapropterin) or Palynziq (pegvaliase) both approved to reduce levels of the amino acid, although they ...

AMT-130 is administered through magnetic resonance imaging-guided, convection-enhanced stereotactic neurosurgical delivery into the striatum.

Liquidia Corporation announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) resubmission for YUTREPIA™ (treprostinil) inhalation powder to treat ...