pharmaphorum9 个月
Synlogic shuts down after PKU drug fails phase 3 trial
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 ...
pharmaphorum4 天
Otsuka buys Jnana and PKU drug in $1.1bn deal
"The addition of Jnana's drug discovery technology and small molecule pipeline in PKU and autoimmune diseases will strengthen our R&D in the Boston area of the US, one of the most important ...
Understanding orphan drugs: what are they, how are they classified and what are their ...
such as Phenylketonuria (PKU) and Maple Syrup Urine Disease (MSUD). Group 3 covers conditions like Gaucher Disease and Pompe ...
FiercePharma11 天
Dr. Reddy's pulls more than 300K bottles of thyroid drug, citing presence of a carcinogen
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.
Investing1 个月
FDA accepts new PKU drug application from PTC Therapeutics
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 ...
Investing1 个月
FDA sets target date for PTC's PKU drug review
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 ...
Investing1 个月
FDA sets target date for PTC's PKU drug review
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 ...
WebMD6 年
Vitamins by Condition
Considering taking supplements to treat phenylketonuria pku? Below is a list of common natural remedies used to treat or reduce the symptoms of phenylketonuria pku. Follow the links to read common ...
Next Generation Gene Therapeutics (NGGT) Announces Positive New Data On NGGT002 For The ...
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 ...
Relief Therapeutics Announces Publication of Plain Language Summary on PKU GOLIKE
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 ...
Relief Therapeutics Reports New Study Results for RLF-OD032 and Files Provisional Patents
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 ...