Pratish Thakore, Ph.D.’s Post

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Scientific Communication | Cardiovascular Biology | Neuroscience & Neurodegeneration

ARMGO Pharma and the National Institutes of Health have collaborated on a Phase 1b study of Rycal ARM210, a treatment for Ryanodine Receptor Type 1 (RyR1) Related Myopathies (RyR1-RM), an orphan muscle disease. RyR1 is an intracellular calcium-release channel vital to muscle contraction. Mutant RyR1 channels cause intracellular calcium leaks that impair muscle contraction, leading to muscle weakness and loss of function. The trial demonstrated that ARM210 is safe and well-tolerated, and it improved muscle strength and alleviated fatigue in patients with RyR1-RM. ARMGO Pharma is hopeful that ARM210 can also be used to treat Catecholaminergic Polymorphic Ventricular Tachycardia, a genetic life-threatening arrhythmia caused by mutations in RyR2. Read more about this development in the treatment of orphan muscle diseases at https://lnkd.in/gxqfTjiw. #RyanodineReceptor #CalciumChannels #RareArrhythmias #MuscleHealth #MedicalResearch

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Ali Divan, Ph.D.

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8mo

Way to repurpose and find additional indications for a relatively rare disease drug.

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