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NewMitochondrial Myopathy – Drugs In Development, 2024
Empower your strategies with our Mitochondrial Myopathy – Drugs In Development, 2024 report and make more profitable business decisions. Mitochondria are known as "the powerhouse of the cell," but when defects occur they give rise to mitochondrial diseases. Mitochondrial diseases affecting the muscles are known as mitochondrial myopathies. These can occur because of fetal gene mutations in the mitochondria, resulting in childhood myopathies. Symptoms include fatigue, muscle weakness, stunted growth, and exercise intolerance, as production of adenosine triphosphate (ATP), a cellular...
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NewLeber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Drugs In Development, 2024
Empower your strategies with our Leber's Hereditary Optic Neuropathy (Leber Optic Atrophy) – Drugs In Development, 2024 report and make more profitable business decisions. Leber’s hereditary optic neuropathy (LHON) is an inherited form of vision loss. This condition usually begins in a person's teens or twenties; rare cases may appear in early childhood or later in adulthood. Males are affected much more often than females. Symptoms include eye pain or discomfort, numbness, tingling, and clouding of vision. Predisposing factors include age,...
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NewFriedreich Ataxia – Drugs In Development, 2024
Empower your strategies with our Friedreich Ataxia – Drugs In Development, 2024 report and make more profitable business decisions. Friedreich’s ataxia (FA) is a neuromuscular disease that mainly affects the nervous system and the heart. FA is a hereditary disease, caused by a defective gene that can be passed down through a family. Signs and symptoms include ataxia, weakness and spasticity, sensory impairment, skeletal abnormalities, cardiac difficulties, and diabetes. The Friedreich Ataxia drugs in development market research report provide comprehensive information...
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NewRetinal Degeneration – Drugs In Development, 2024
Empower your strategies with our Retinal Degeneration – Drugs In Development, 2024 report and make more profitable business decisions. Retinal degeneration refers to a group of diseases that cause progressive damage to the cells of the retina, the light-sensitive tissue at the back of the eye crucial for vision. These conditions lead to a gradual loss of vision, which can range from mild to severe and can eventually result in blindness. There are several types of retinal degenerative diseases, including: Retinitis...
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NewDuchenne Muscular Dystrophy – Drugs In Development, 2024
Empower your strategies with our Duchenne Muscular Dystrophy – Drugs In Development, 2024 report and make more profitable business decisions. Duchenne muscular dystrophy (DMD) is a genetic disorder that causes muscles to gradually weaken over time. Signs and symptoms of DMD include fatigue, learning difficulties, intellectual disability, muscle weakness, and progressive difficulty walking. Risk factors include gender and family history. Treatments include steroid medication, respiratory therapy, and surgery. The Duchenne Muscular Dystrophy drugs in development market research report provide comprehensive information...
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NewRecessive X Chromosome Disorders – Drugs In Development, 2024
Empower your strategies with our Recessive X Chromosome Disorders – Drugs In Development, 2024 report and make more profitable business decisions. A recessive X chromosome disorder, also known as an X-linked recessive disorder, is a genetic condition caused by a mutation on one of the X chromosomes, one of the two sex chromosomes. Typically, males have one X and one Y chromosome (XY), while females have two X chromosomes (XX). In recessive X-linked disorders, the mutated gene is located on the...
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NewLikelihood of Approval and Phase Transition Success Rate Model – IXC-103 in Friedreich Ataxia
Empower your strategies with our Likelihood of Approval and Phase Transition Success Rate Model - IXC-103 in Friedreich Ataxia report and make more profitable business decisions.This report provides you with the data that allows you to track and predict the specific likelihood of approval (LOA) and phase transition success rate (PTSR) of a drug using GlobalData’s proprietary machine learning algorithms developed using over 10 years of historical data.IXC-103 in Friedreich AtaxiaDrug Details:IXC-103 (dimethyl fumarate) is under development for the treatment...
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NewLikelihood of Approval and Phase Transition Success Rate Model – ETH-155008 in Relapsed Chronic Lymphocytic Leukemia (CLL)
Empower your strategies with our Likelihood of Approval and Phase Transition Success Rate Model - ETH-155008 in Relapsed Chronic Lymphocytic Leukemia (CLL) report and make more profitable business decisions.This report provides you with the data that allows you to track and predict the specific likelihood of approval (LOA) and phase transition success rate (PTSR) of a drug using GlobalData’s proprietary machine learning algorithms developed using over 10 years of historical data.ETH-155008 in Relapsed Chronic Lymphocytic Leukemia (CLL) Drug Details:ETH-155008 is...