Detailed study results will be presented at an upcoming medical meeting and submitted to health authorities later this year to expand Palynziq’s label to include adolescents.

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 ...

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 ...

I am truely greatful for the opportunity to share my PKU journey and my experience with Palynziq. Today, I’m off to Greensboro, North Carolina, to meet others and hopefully encourage others with ...

RBC Capital analyst Luca Issi maintained a Hold rating on BioMarin Pharmaceutical (BMRN – Research Report) on April 3 and set a price target of ...

At close: April 7 at 4:00:01 PM EDT ...

4 April 2025 A planned autumn review of the UK’s 2024 Voluntary Scheme for Branded Medicines Pricing, Access, and Growth (VPAG) will be brought forward to report back in June, it has been announced.