Amino Acid Metabolism Disorders is an indication for drug development with over 70 pipeline drugs currently active. According to GlobalData, preregistered drugs for Amino Acid Metabolism Disorders have a 90% likelihood of approval (LoA) indication benchmark. GlobalData’s report assesses how phase transition success rate (PTSR) and likelihood of approval (LoA) scores for pipeline drugs in Amino Acid Metabolism Disorders compared to historical benchmarks. Buy the report here.
GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.
Amino Acid Metabolism Disorders overview
Amino acids are building blocks of proteins and enzymes. Metabolism of amino acids results in energy production and other mechanisms. Any disturbance in this pattern results in metabolic disorders. These are a group of inherited diseases. Amino acid metabolic disorders include Phenylketonuria, maple syrup urine disease, citrullinemia (CIT), ornithine Transcarbamylase deficiency, and argininosuccinate lyase (ASL) deficiency. All these conditions are genetically inherited autosomal /X-linked recessive. Diagnosis is done by lab examination of urine, blood analysis, and biochemistry reports. Low protein diet is recommended and in severe cases liver transplant is the option.
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For a complete picture of PTSR and LoA scores for drugs in Amino Acid Metabolism Disorders, buy the report here.
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