Navigating the Neurological Maze: McGill’s Breakthrough Study Explores SCA6 and Endosomal Dysfunction
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Navigating the Neurological Maze: McGill’s Breakthrough Study Explores SCA6 and Endosomal Dysfunction

What role do brain alterations play in the onset and progression of spinocerebellar ataxia type 6 (SCA6)? While previous studies highlighted cerebellar changes in individuals with SCA6, the underlying mechanisms…

Continue Reading Navigating the Neurological Maze: McGill’s Breakthrough Study Explores SCA6 and Endosomal Dysfunction
Ruxoprubart Granted Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH)
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Ruxoprubart Granted Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH)

Developing novel therapeutics for rare diseases is increasingly important. Understanding the unmet needs of rare disease communities and providing effective, accessible therapies can significantly improve the lives of those affected.…

Continue Reading Ruxoprubart Granted Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH)