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Product Insights
Alpha N-Acetylglucosaminidase (N Acetyl Alpha Glucosaminidase or NAGLU or EC 3.2.1.50) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Alpha N-acetylglucosaminidase Pipeline Products Market Report Overview Alpha N-acetylglucosaminidase is a protein associated with Sanfilippo syndrome. This enzyme is located in lysosome compartments within cells that digest and recycle different types of molecules. Alpha N-acetylglucosaminidase is involved in the stepwise breakdown of large molecules called glycosaminoglycans (GAGs). Alpha N-acetylglucosaminidase removes a sugar called N-acetylglucosamine when it is at the end of the GAG chain. The Alpha N-Acetylglucosaminidase pipeline market research report provides comprehensive information on the therapeutics under development for...
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Likelihood of Approval and Phase Transition Success Rate Model – AGT-181
Overview How likely is it that a drug will get approved? Will the drug transition to the next phase of its clinical pathway? This report provides you with the data to allow you to track and predict specific likelihood of approval (LOA) and phase transition success rate (PTSR) of a drug using a combination of machine learning and proprietary models. Likelihood of Approval (LoA) - Industry AGT-181 Drug Details AGT-181 (HIRMAb-IDUA) is an investigational enzyme replacement therapy (ERT) for the...
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Net Present Value Model: Evazarsen Sodium
Overview Evaluating the value of drugs is a complicated practice and requires a deep knowledge of the drug itself, the market currently and in the future, knowledge of cash inflows and outflows and the potential success rates for each stage of drug development. GlobalData has done all of this work for you, leveraging its gold standard Drugs Intelligence database to create high-value NPV models for purchase on a drug-by-drug basis. Drug Operating Profit Model Evazarsen Sodium Drug Details IONIS-AGT-LRx is...
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Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Mucopolysaccharidosis type III (MPS III) is also known as Sanfilippo syndrome. It is an inherited metabolic disease caused by the absence or malfunctioning of certain enzymes needed to break down molecules called glycosaminoglycans (GAG). Symptoms include seizures, hyperactivity, liver and spleen enlargement, severe diarrhea or constipation, and enlargement of tonsils and adenoids. Treatment includes enzyme replacement therapy (ERT). The MPS III pipeline market research report provides comprehensive information on the therapeutics under development for MPS III, complete with analysis by...
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Net Present Value Model: Zilebesiran
Overview Evaluating the value of drugs is a complicated practice and requires a deep knowledge of the drug itself, the market currently and in the future, knowledge of cash inflows and outflows and the potential success rates for each stage of drug development. GlobalData has done all of this work for you, leveraging its gold standard Drugs Intelligence database to create high-value NPV models for purchase on a drug-by-drug basis. Drug Operating Profit Model Zilebesiran Drug Details Zilebesiran sodium (ALNAGT-01)...
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Metachromatic Leukodystrophy (MLD) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Metachromatic leukodystrophy (MLD) is an inherited disorder characterized by the accumulation of fats called sulfatides in cells. The Metachromatic Leukodystrophy pipeline market research report provides comprehensive information on the therapeutics under development for Metachromatic Leukodystrophy, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history, and the latest news and press releases. Additionally,...
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Likelihood of Approval and Phase Transition Success Rate Model – tonlamarsen sodium
Overview How likely is it that a drug will get approved? Will the drug transition to the next phase of its clinical pathway? This report provides you with the data to allow you to track and predict specific likelihood of approval (LOA) and phase transition success rate (PTSR) of a drug using a combination of machine learning and proprietary models. Likelihood of Approval (LoA) - Industry tonlamarsen sodium Drug Details ION-904 is under development for treatment of resistant hypertension. It...
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Likelihood of Approval and Phase Transition Success Rate Model – evazarsen sodium
Overview How likely is it that a drug will get approved? Will the drug transition to the next phase of its clinical pathway? This report provides you with the data to allow you to track and predict specific likelihood of approval (LOA) and phase transition success rate (PTSR) of a drug using a combination of machine learning and proprietary models. Likelihood of Approval (LoA) - Industry evazarsen sodium Drug Details IONIS-AGT-LRx is under development for treatment-resistant hypertension, mild hypertension and...
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Likelihood of Approval and Phase Transition Success Rate Model – zilebesiran sodium
Overview How likely is it that a drug will get approved? Will the drug transition to the next phase of its clinical pathway? This report provides you with the data to allow you to track and predict specific likelihood of approval (LOA) and phase transition success rate (PTSR) of a drug using a combination of machine learning and proprietary models. Likelihood of Approval (LoA) - Industry zilebesiran sodium Drug Details Zilebesiran sodium (ALNAGT-01) is under development for hypertension. The drug...
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Product Insights
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
MPS I (Mucopolysaccharidosis I) is an inherited lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). Symptoms include abnormal bones in the spine, claw hand, cloudy corneas, deafness and heart valve problems. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy. The Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) Pipeline market research report provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis I...