BioMarin to Acquire Rights to Phenylketonuria (PKU) Franchise From Merck Serono
Strengthens and Expands BioMarin's Leadership Position Serving PKU Patients with Rights to Both Kuvan(R) (sapropterin dihydrochloride) and Pegvaliase Beyond North America 2016 Full-year Guidance for Kuvan Expected to be Between $320M-$350M including $70... Biopharmaceuticals, AcquisitionsBioMarin Pharmaceutical, Merck Serono, Kuvan, Phenylketonuria
ConclusionTreatment for PKU incurs a substantial time and cost burden on persons with PKU and their families. Estimated medical expenditures using claims data varied by age group, but sapropterin represented the largest cost for PKU treatment from a payer perspective across age groups.
Phenylketonuria is a hereditary metabolic disorder due to the deficiency of tetrahydrobiopterin or phenylalanine hydroxylase. Delayed diagnoses of it manifest a progressive irreversible neurological impairment in the early years of the disease. Guthrie test and tandem mass spectrometry aided in early detection and intervention of phenylketonuria, which significantly decreased the disability of patients as well as reducing the need for diagnosis in adults. This is a case report of a 60-year-old Asian man, characterized by severe visual-spatial disorders and bilateral diffuse symmetric white matter lesions on magnetic resona...
Analyst, 2019, Accepted Manuscript DOI: 10.1039/C9AN01642B, PaperJennifer Wild, Meera Shanmuganathan, Mika Hayashi, Murray Potter, Philip Britz-McKibbin Management of phenylketonuria (PKU) requires lifelong restriction of phenylalanine (Phe) intake using specialized medical foods to prevent neurocognitive impairment in affected patients. However, dietary adherence is challenging to maintain while... The content of this RSS Feed (c) The Royal Society of Chemistry
Condition: Phenylketonurias Intervention: Drug: RTX-134 Sponsor: Rubius Therapeutics Recruiting
Condition: Phenylketonuria Interventions: Drug: CDX 6114; Other: Matching Placebo Sponsor: Nestlé Recruiting