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Genescience Pharmaceuticals describes new PAH stabilizers for PKU
17 Oct 2025 15:18 GMT
Endocrine/metabolic
Genescience Pharmaceuticals Co. Ltd. has identified phenylalanine … to be useful for the treatment of phenylketonuria (PKU).
BioWorld Science Endocrine…
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National PKU Alliance Grant Advances Engineered Native Bacteria Candidate for Phenylketonuria (PKU)
16 Oct 2025 11:00 GMT
… (ENB) candidate for the treatment of Phenylketonuria (PKU), a rare genetic disorder that … to expand access to treatments, accelerate the development of new therapies and … innovative approach holds promise for treating—and potentially curing—a wide …
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Consistency in the Chaos: FDA Approvals Within Average Range as Q4 Kicks Off
13 Oct 2025 05:23 GMT
… month.
As for rejected drug applications, these were … recessive dystrophic epidermolysis bullosa and phenylketonuria. On the other hand … both BioSpace and Jefferies. Treatments for infectious diseases and … term, the FDA approved 46 new drugs—more than double …
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Following Cancelled FDA Meeting, Kezar To Cut Employees, Eyes Strategic Alternatives
09 Oct 2025 01:59 GMT
… four trial participants with AIH from continuing treatment in … develop next-generation antibody-drug conjugate (ADC) therapies to treat … , Rising Pharma is focused on prescription generic drugs, with … the liver, such as phenylketonuria and urea cycle disorders …
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Modified Sijunzi Decoction Inhibits Pancreatic Cancer Progression and Improves Immune Suppression: Network Pharmacology and Experimental Validation
02 Oct 2025 06:38 GMT
… macrophage, and dendritic cell) development, activation, and function.61 … in treating digestive malignant tumor. Chin J Integr Med. 2007 … the treatment of colorectal cancer based on network pharmacology and … phase I clinical trial. Invest New Drugs. 2018;36(3 …
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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 Treat Phenylketonuria (PKU) and Chronic Kidney Disease (CKD)
11 Sep 2025 11:30 GMT
… stage biopharmaceutical company developing small molecule precision … endpoints included pharmacokinetics, food effect, pharmacodynamic measures of … events and no treatment-related adverse events … the potential to treat both phenylketonuria (PKU) and chronic …
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Investigating the Mechanism of Jiawei Weijin Decoction in Treating Non-Small Cell Lung Cancer Using Network Pharmacology, Bioinformatics Analysis and Experimental Validation
25 Sep 2025 08:23 GMT
… natural drugs to inhibit the target of SPP1 in treating the … on combination and versus drug therapies. Biomed Pharmacother. 2021;138:111350. … and reducing errors. Stat Med. 1996;15(4):361– … of osteopontin in cancer development and treatment. Heliyon. 2023;9(10 …
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Vanguard, BlackRock, and Armistice Capital Maintain Large Stakes in PTC Therapeutics Amid Pipeline Developments
16 Sep 2025 23:15 GMT
… , when the FDA approved Sephience (sepiapterin) to treat phenylketonuria (PKU) across all disease … Palynziq have long dominated pharmacologic treatment for PKU. But Reuters reported … large positions amid these developments are consistent with index …
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Maze Therapeutics Drug Shows Potential to Top Asset Acquired by Otsuka for $800M
14 Sep 2025 18:59 GMT
… treatment-related complications leading to discontinuation of the study drug … Maze aims to treat is phenylketonuria, in which … . BioMarin Pharmaceutical markets two FDA-approved medications for the … which is in development for treating patients with APOL1 …
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Maze reports positive results from Phase I trial of PKU and CKD treatment
12 Sep 2025 17:19 GMT
Maze Therapeutics has shared positive outcomes from the randomised Phase I study of the oral SLC6A19 inhibitor, MZE782, for treating phenylketonuria (PKU) and chronic kidney disease (CKD).
The placebo-controlled, double-blind trial, which involved 112 …
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