National PKU Alliance Grant Advances Engineered Native Bacteria Candidate for Phenylketonuria (PKU)

The company is advancing a robust pipeline of ENB-based therapeutics targeting both rare and prevalent chronic diseases, ...
Morningstar

Maze Therapeutics Announces Positive First-in-Human Results from Phase 1 Trial of MZE782, Establishing Proof of Mechanism for a Potent, Oral SLC6A19 Inhibitor with Potential to ...

Phase 1 data in healthy volunteers exceed expectations and support best-in-class potential, enabling Phase 2 advancement for both intended indications of PKU and CKD Dose-dependent urinary amino acid ...
Insider Monkey

BioMarin Pharmaceutical Inc.’s (BMRN) Phenylketonuria Candidate Drug Impresses in Phase 3 Trials

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the best beginner stocks to buy, according to analysts. On September 6, the company delivered positive pivotal Phase 3 data for PALYNZIQ ...
Independent.ie

'Everything has to be planned or thought through' – Wicklow dad on raising two sons with PKU

When Conor Byrne got the call from the hospital just days after his son Jacob was born, he knew something wasn’t right. “Don’t Google it,” they were told — words that no new parent ever wants to hear.

Can BioMarin (BMRN) Turn PALYNZIQ Success Into a Stronger Rare Disease Growth Story?

In September 2025, BioMarin Pharmaceutical Inc. was removed from the FTSE All-World Index following share price pressure, despite announcing positive Phase 3 results for its PALYNZIQ® therapy in teens ...
Euractiv

Cracking the ‘unmet medical need’ code, Gastein experts weigh in

EMA’s Steffen Thirstrup suggests replacing 'medical' with 'therapeutic' but patient advocate Tobias Hagedorn warns that ...