Shares in Synlogic have halved in value after the company reported that a phase 3 trial of its main pipeline drug for rare metabolic disorder phenylketonuria (PKU) failed to show efficacy ...

such as Phenylketonuria (PKU) and Maple Syrup Urine Disease (MSUD). Group 3 covers conditions like Gaucher Disease and Pompe ...

has accepted for filing its New Drug Application (NDA) for sepiapterin, a treatment designed for both pediatric and adult patients with phenylketonuria (PKU). The company expects to receive a ...

An 11-year-old girl with the rare disease phenylketonuria (PKU) has lost a legal battle in the High Court over a decision by NICE to deny NHS access to the only approved drug in the UK.

This drug is intended for the treatment of phenylketonuria (PKU) in both pediatric and adult patients, covering all age groups and disease subtypes. The company also announced the recent ...

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 ...

This drug is intended for the treatment of phenylketonuria (PKU) in both pediatric and adult patients, covering all age groups and disease subtypes. The company also announced the recent publication ...

The patents, based on these new findings, cover additional claims for Relief’s investigational drug RLF-OD032, a highly concentrated, novel liquid formulation of sapropterin dihydrochloride, for the ...

The U.S. unit of Indian drugmaker Dr. Reddy’s Laboratories is recalling 331,590 bottles of cinacalcet tablets used to treat hyperparathyroidism due to the presence of a known carcinogen. | The U.S.

New Study Results Indicate Superior Absorption of RLF-OD032 in Fasted State Compared to KUVAN®, Potentially Enabling Flexible Dosing Options GENEVA, SWITZERLAND / ACCESSWIRE / October 25, 2024 /RELIEF ...

GENEVA, SWITZARLAND / ACCESSWIRE / October 29, 2024 /RELIEF THERAPEUTICS Holding SA (SIX:RLF)(OTCQB:RLFTF)(OTCQB:RLFTY) (Relief, or the Company), a biopharmaceutical company committed to delivering ...

in treating phenylketonuria (PKU). The recent acceptance of the company’s New Drug Application (NDA) by the FDA signals a positive regulatory momentum. Additionally, the existing safety profile ...