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Eton Pharmaceuticals Announces FDA Acceptance of New Drug Application for ET-600 (Desmopressin Oral Solution)
08 Jul 2025 10:50 GMT
… pharmaceutical company focused on developing and commercializing treatments for … . Food and Drug Administration (FDA) and has … ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine … of discovering, developing and commercializing drugs that are …
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Sephience™ (sepiapterin) Granted Marketing Authorization by the European Commission for the Treatment of Children and Adults Living with Phenylketonuria (PKU)
23 Jun 2025 12:16 GMT
… treatment of children and adults living with phenylketonuria (PKU). … trial as well as evidence of durable treatment … new drug in the United States.
About Phenylketonuria
Phenylketonuria (PKU) … As with any pharmaceutical under development, there are significant …
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Agios Appoints Dr. Jay Backstrom to Board of Directors
08 Jul 2025 11:00 GMT
… of Research and Development at Acceleron Pharma, which was acquired … (MDS)-associated anemia and phenylketonuria (PKU). In addition to its … siRNA as a potential treatment for polycythemia vera. For … review boards at clinical trial sites and publication review …
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Timely diagnosis crucial for treatment of PKU
29 Jun 2025 11:56 GMT
… phenylketonuria (PKU) do not initially have any symptoms, the first step in treating PKU …
PKU was discovered in the 1930s. The initial treatment for it was developed … of these diseases have effective drug treatment available.
According to the World …
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Biotech Stocks Facing FDA Decision In July 2025
29 Jun 2025 07:25 GMT
… drugs in the U.S. for treating … .
Apellis Pharmaceuticals Inc. (APLS)
Apellis Pharma';s … disability, seizures, developmental delays, memory impairment, … drug Kuvan and injectable drug Palynziq are the two FDA-approved treatments for phenylketonuria …
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Phenylketonuria (PKU): Market View: 262 Molecules In The Pipeline With 18 Assets Nearing Market
18 Jun 2025 10:10 GMT
… and future competitive landscape in Phenylketonuria (PKU) Therapeutics. In 2024, there … period. Reasons to Buy Develop and design your in- … Treatment Overview
4 Marketed Drugs Assessment
4.1 Leading Marketed Drugs
… Top 20 countries for Trial Sites
7.14 Top …
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Eton Pharmaceuticals Announces Addition to Russell 2000® and Russell 3000® Indexes
27 Jun 2025 10:50 GMT
… innovative pharmaceutical company focused on developing and commercializing treatments for rare … INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous … process of discovering, developing and commercializing drugs that are safe …
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A Systematic Review of SNPs Screening for Platinum-Related Pharmacodynamics and Pharmacokinetics Genes in Non-Small Cell Lung Cancer for Precision Medicine
26 Jun 2025 22:33 GMT
… promising efficacy in treating NSCLC. In 1995 … medication development for NSCLC, guided by genetic profiling for personalized treatment … old drugs, new integrated perspective. Biomed Pharmacother. 2020 … flow assay for phenylketonuria diagnostic. Anal Chem …
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Germany will be first launch for PTC's phenylketonuria drug
25 Jun 2025 15:28 GMT
… Therapeutics' treatment for the rare disease phenylketonuria (PKU), Sephience, setting … and some patients need drug therapies to control Phe levels … first for the drug, although, the FDA in the US … living with PKU in world markets where the drug can be …
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PTC Therapeutics’ Sephience gets European marketing authorization to treat children and adults living with phenylketonuria
24 Jun 2025 08:38 GMT
… the treatment of children and adults living with phenylketonuria (PKU). … acts as a dual pharmacological chaperone (sepiapterin and … new drug in the United States.
Phenylketonuria (PKU) is … disability, seizures, delayed development, memory loss, and …