… innovative pharmaceutical company focused on developing and commercializing treatments for … product used to treat an ultra-rare … ®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine … of discovering, developing and commercializing drugs that are …

… syndrome, such as developmental delay and growth … Ascendis Pharma’s investigational achrondroplasia drug TransCon … Phase II ACcomplisH trial, treatment improved annualized growth … drug to include adolescents from 12 through 17 years with phenylketonuria (PKU …

… buying this biotech stock. BioMarin Pharmaceutical';s (BMRN 0.79% … , the U.S. Food and Drug Administration (FDA) set a PDUFA date of … in the treatment of adolescents ages 12 through 17 with phenylketonuria. European Union …

… developing contexts.10 At the same time, informal self-treatment … http://charls.pku.edu.cn). Ethical Approval … month? (Excluding prescription medications)” Outpatient care was assessed … mortgage borrowing, and medication adherence. Soc Sci Med. 2022;314: …

… treatment adoption. Data from the report underscores the impact of orphan drug development … Pompe disease, and phenylketonuria informing MPS strategies. Government … Pharmaceutical Company Limited, Sumitomo Pharma Co., Ltd., Ultragenyx Pharmaceutical …

… for the treatment of adult and pediatric patients with phenylketonuria (PKU). Sephience is … the potential to treat a broad range of PKU patients. Sephience is … such factors. As with any pharmaceutical under development, there are significant risks …

… FDA pathway provides a way for these types of drugs … focus on treating a metabolic disorder called phenylketonuria, or PKU, that … early treatment and monitoring, PKU can hinder brain development and impair … new drug, each requiring its own clinical trial. But …

… exorbitant costs of developing gene-based treatments for them make … the eye of Food and Drug Administration leaders. Last November, … the first platform to treat the types of mutations … system by starting with phenylketonuria, or PKU. The disease is caused …

… Spain, diagnosed with phenylketonuria (PKU) through newborn screening, … clinicians should consider pharmacologic treatment discontinuation in patients with … after treatment initiation, including the development of class … and to her medication. Her blood Phe …

… received a BCMA antibody-drug conjugate. J Clin Oncol … also approved by the FDA as a companion … treatment of hyperphenylalaninemia in patients aged ≥1 month with sepiapterin-responsive phenylketonuria … open-label extension trial. Genet Med. 2024;26:101138 …