HSE bosses have advised people to apply for a little-known health scheme that thousands are eligible for. The Long-Term ...

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Our chief medical officer, Professor Jonathan Benger, explains the crucial role the HST programme plays in encouraging ...

Maze has reached a pivotal moment in our journey. On the heels of a successful IPO and with two ongoing clinical-stage programs – MZE829 for ...

evaluating the potential meaningful benefits of sepiapterin treatment in phenylketonuria or PKU patients. Sepiapterin has a dual mechanism of action to increase activity of the phenylalanine ...

Phenylketonuria (PKU) is a disorder where an amino acid called phenylalanine accumulates inappropriately in the body. Phenylalanine is a protein building block the body obtains through the ...

A rare disease in the US is one in 1,500, or fewer than 200,000 people, and an ultra-rare disease is one in 50,000 or fewer ...

PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics ...

MZE829 Phase 2 HORIZON Trial Enrolling Patients with APOL1 Kidney Disease (AKD); Initial Data Expected in Q1 2026MZE782 Phase 1 Healthy Volunteer ...

Korea Investment CORP grew its position in shares of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN – Free Report) by 362.5% ...

Learn more about whether Alvotech or PTC Therapeutics, Inc. is a better investment based on AAII's A+ Investor grades, which ...

Synthetic biology specialist Synlogic will start phase 3 testing of a drug for rare metabolic disorder phenylketonuria (PKU) next year, after reporting solid proof-of-concept data.