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Phenylketonuria as an Adherence Disease
12 Apr 2025 16:27 GMT
… aspects of PKU treatment: diet, medications, and … , using drugs such as … drug adherence rates among patients with seven different medical conditions. Pharmacotherapy … development of executive functions and social cognition in children with phenylketonuria …
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Key Driver In The Phenylketonuria PKU Global Market Report 2025: Increasing Cases Of Genetic Disorders Driving The Size
27 Mar 2025 14:44 GMT
… product developed with patented, pharmaceutical-grade Physiomimic technology for dietary management of phenylketonuria PKU … : Moderate PKU with medication, Variant PKU with treatment variations
4 By Classic Phenylketonuria PKU: Classic PKU with …
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BioMarin’s trial for phenylketonuria therapy meets primary efficacy endpoint
03 Apr 2025 11:56 GMT
… phenylketonuria (PKU) against only diet.
The adolescent individuals in the trial … According to BioMarin Pharmaceutical, the trial is being carried … down Phe.
The treatment involves a dosing regimen … chief research and development officer and executive vice …
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BioMarin Pharma’s phase 3 PEGASUS trial evaluating Palynziq in adolescents with phenylketonuria meets primary efficacy endpoint
03 Apr 2025 13:20 GMT
… aged 12-17 with phenylketonuria (PKU) compared to diet … approved to treat adults with PKU.
Detailed results … pharmacokinetics.
The study is being conducted in two parts: the primary treatment … can prevent major developmental neurological toxicities, but …
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Eton Pharmaceuticals Out-Licenses International Rights to Increlex®
01 Apr 2025 11:00 GMT
… an innovative pharmaceutical company focused on developing and commercializing treatments for rare … INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine … process of discovering, developing and commercializing drugs that are safe …
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Metabolomics Analysis of the Mechanism of Yuquan Capsules in the Treatment of T2DM Rats Using UPLC/MS
27 Mar 2025 06:01 GMT
… a collection of Yuquan Pills (YQP) in “Wanbinghuichun … clinical manifestation of phenylketonuria (PKU).25,26 We … pharmacochemistry of Chinese medicine. China J Chin Mater Med … Jerry V. Phenylketonuria: current treatments and future developments. Drugs. 2019;79 …
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Agios Pharmaceuticals develops PAH mutant stabilizers for phenylketonuria
26 Mar 2025 19:14 GMT
Endocrine/metabolic
Agios Pharmaceuticals Inc. has patented new 4- … to be useful for the treatment of phenylketonuria.
BioWorld Science Endocrine…
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Phenylketonuria Treatment Market Set to Surge at 7.8% CAGR, to Reach US$ 1.62 Billion by 2034: Fact.MR Report
20 Mar 2025 06:48 GMT
… These developments enhance patient compliance and treatment … first oral treatment for phenylketonuria (PKU) of its … flagship phenylketonuria program and small drug … channel (hospital pharmacies, retail pharmacies, online pharmacies, hypermarkets …
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iECURE Announces Presentation of Full Data for the First Infant Dosed with ECUR-506 in OTC-HOPE Phase 1/2 Clinical Trial at the 2025 ACMG Annual Clinical Genetics Meeting
21 Mar 2025 12:44 GMT
… development of mutation-agnostic in vivo gene insertion therapies for the treatment … 506 dosing, ammonia scavenger medication was discontinued based on … first-in-human clinical trial of ECUR-506 in … will also assess the pharmacokinetics and efficacy of ECUR- …
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PTC Therapeutics Reveals New Sepiapterin Data From Phase 3 Study In Patients With Inherited Disorder, FDA Decision Expected In July
20 Mar 2025 16:37 GMT
… from the Phase 3 APHENITY trial and subsequent open-label extension … benefits of sepiapterin treatment for the full spectrum of phenylketonuria (PKU) patients.
Also … (GPV) consistent with classical PKU.
PTC submitted FDA regulatory approval applications in …
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