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Showing posts from October, 2024

FDA Approves AbbVie's VYALEV for 24-Hour Treatment of Advanced Parkinson's Disease

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  AbbVie announced that the U.S. FDA has approved VYALEV™ (foscarbidopa and foslevodopa) as the first and only subcutaneous 24-hour infusion of levodopa-based therapy for treating motor fluctuations in adults with advanced Parkinson’s disease (PD). Robert A. Hauser, M.D., MBA, Professor of Neurology and Director of the Parkinson’s and Movement Disorder Center at the University of South Florida, highlighted that this new, non-surgical regimen provides continuous delivery of levodopa morning, day and night. Publish Date: 17-10-2024   Source: AbbVie Parkinson’s disease, a chronic and progressive disorder of the central nervous system, falls under the category of motor system disorders. This condition stems from the loss of cells in the substantia nigra region of the brain, which are responsible for producing dopamine, a crucial neurotransmitter facilitating communication within the brain. Dopamine ensures the smooth and coordinated operation of the body’s muscles and mov...

Sangamo Therapeutics Gains FDA Pathway to Accelerated Approval for ST-920 Gene Therapy in Fabry Disease Disease

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  Sangamo Therapeutics, Inc., a genomic medicine company, announced the outcome of a recent successful interaction with the U.S. Food and Drug Administration. This interaction has provided a clear regulatory pathway to accelerated Approval for isaralgagene civaparvovec (ST-920), its wholly owned gene therapy product candidate for the treatment of Fabry disease. “Fabry disease is a debilitating condition with a significant unmet medical need,” said Sandy Macrae, CEO of Sangamo Therapeutics. Publish Date: 22-10-2024   Source: Sangamo Therapeutics, Inc. Fabry disease, a rare genetic metabolic storage disorder, results in the progressive failure of organs and premature death. It arises from a mutation in the galactosidase alpha gene, causing a deficiency or absence of α-galactosidase A (GALA), a lysosomal enzyme. Consequently, this deficiency impairs the breakdown of globotriaosylceramide (Gb3) and related glycosphingolipids, gradually accumulating Gb3 and glycosphingolip...

FDA Grants Orphan Drug Designation to Remedy Pharma's CIRARA for Treatment of Acute Ischemic Stroke

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  Remedy Pharmaceuticals, a pioneer in stroke drug development, has announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Division (OOPD) has granted Orphan Drug Designation to CIRARA for treating large territory acute ischemic stroke, including large hemispheric infarctions (LHI). According to Sven Jacobson, CEO of Remedy Pharmaceuticals, this designation highlights the unique mechanisms and universally poor outcomes of large territory ischemic strokes compared to smaller strokes. Publish Date: 22-10-2024   Source: Remedy Pharmaceuticals Acute ischemic stroke (AIS) occurs when a blood vessel supplying the brain becomes obstructed, resulting in a significant reduction or cessation of blood flow to the affected area. Stroke stands as one of the foremost contributors to mortality and disability across the globe. Modifiable risk factors encompass a range of health conditions and lifestyle factors that individuals have the potential to influ...