DelveInsight’s, “Phenylketonuria Pipeline Insight 2023” report provides comprehensive insights about 10+ companies and 15+ pipeline drugs in the Phenylketonuria pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Key takeaways from the Phenylketonuria Pipeline Report
Recent Developmental Activities in the Phenylketonuria Pipeline
Get an overview of the Phenylketonuria Pipeline landscape @ Phenylketonuria Ongoing Clinical Trials Analysis
Phenylketonuria Overview
Phenylketonuria (PKU) is an inborn error of metabolism that is detectable during the first days of life via routine newborn screening. PKU is inherited in an autosomal recessive pattern. PKU is characterized by absence or deficiency of an enzyme called phenylalanine hydroxylase (PAH), responsible for processing the amino acid phenylalanine. Infants with PKU typically appear normal at birth. With early screening and dietary treatment, affected individuals may never show symptoms of PKU. Symptoms may include vomiting, irritability, and/or a red skin rash with small pimples. Neurological symptoms are present in some untreated patients with PKU, including seizures, abnormal muscle movements, tight muscles, increased reflexes, involuntary movements, or tremor. The goal of treatment for PKU is to keep plasma phenylalanine levels within 120-360 umol/L (2-6 mg/dL). This is generally achieved through carefully planned and monitored diet.
Phenylketonuria Emerging Drugs Profile
Phenylketonuria Pipeline Report
There are approx. 10+ key companies which are developing the therapies for Phenylketonuria. The companies which have their Phenylketonuria drug candidates in the most advanced stage, i.e. Phase III include PTC Therapeutics.
For further information, refer to the detailed Phenylketonuria pipeline report @ Phenylketonuria Treatment Landscape
Scope of the Phenylketonuria Pipeline Report
Table of Content
Got Queries? Find out the related information on Phenylketonuria Mergers and acquisitions, Phenylketonuria Licensing Activities @ Phenylketonuria Emerging Drugs, and Recent Trends
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