Mechanisms of pathogenesis and molecular targets in spinal and bulbar muscular atrophy

Published on June 30, 2016   19 min
0:00
Hello and welcome to all listeners, my name is Carlo Rinaldi, I'm a Neurologist at University of Oxford. In the next 30 minutes or so, I'll be discussing the mechanism of pathogenesis and molecular targets in a disease called spinal and bulbar muscular atrophy or SPMA.
0:18
I will first give some background on when and how the disease was discovered, followed by the etiology and an update regarding the most recent understanding of the disease pathogenesis. I will then talk about the main histological and clinical features which helps clinicians with the diagnosis and which are the diseases that most frequently SPMA is diagnosed with. I will then discuss some of the most promising therapeutic strategies currently being tested in preclinical studies in animal models and in clinical trials.
0:51
SPMA goes under in many names, Kennedy's disease, X-linked spinal muscular atrophy type 1, X-linked spinal and bulbar muscular atrophy, spinal bulbar muscular atrophy, and spinal and bulbar muscular atrophy just to name some. Kennedy's disease comes from the name of the neurologist that described the disease in the late '60s.
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Mechanisms of pathogenesis and molecular targets in spinal and bulbar muscular atrophy

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