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Product Insights
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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Product Insights
Globoid Cell Leukodystrophy (Krabbe Disease) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Krabbe disease also called globoid cell leukodystrophy is a degenerative disorder caused by the deficiency of an enzyme called galactosylceramidase. This enzyme deficiency impairs the growth and maintenance of myelin. Symptoms include seizures, Muscle spasms, Loss of head control, vomiting, fevers, irritability, and excessive crying. Risk factors include family history. Treatment focuses on managing symptoms (anticonvulsant, muscle relaxer) and providing supportive care. The Globoid Cell Leukodystrophy (Krabbe Disease) pipeline market research report provides comprehensive information on the therapeutics under development...
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Mucopolysaccharidosis II (MPS II) (Hunter Syndrome) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is a condition that affects many different parts of the body and occurs almost exclusively in males. Signs and symptoms include claw-like hands, protruding tongue, changing facial features, including thickening of the lips, tongue and nostrils and delayed development. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy. The Mucopolysaccharidosis type II (MPS II) (Hunter Syndrome) pipeline market research report  provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis...
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Sandhoff Disease (Jatzkewitz-Pilz Syndrome) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Sandhoff disease is an inherited lipid storage disorder that progressively destroys nerve cells (neurons) in the brain and spinal cord. It is caused by a deficiency of the enzyme beta-hexosaminidase. Symptoms include progressive nervous system deterioration, problems initiating and controlling muscles and movement, increased startle reaction to sound, early blindness, seizures, spasticity, and myoclonus. Treatment includes anticonvulsants to control seizures. The Sandhoff Disease - Drugs in Development research report provides a comprehensive overview on the therapeutics under development for Sandhoff...
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Batten Disease Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Batten disease, a rare genetic disorder, belongs to a group of progressive degenerative neurometabolic disorders known as neuronal ceroid lipofuscinoses. Symptoms include vision loss, lack of muscle coordination, mental retardation or decreasing mental function, emotional disturbances, seizures, muscle spasms, deterioration of muscle tone, and movement problems. The Batten Disease Drugs in Development market research report provides an overview of the Batten Disease pipeline landscape. The report provides comprehensive information on the therapeutics under development for Batten Disease, complete with analysis...