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She is a physician assistant, TEDx speaker, Inc 5000 honoree, and a thyroid and hormone expert by way of being a patient
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Apitegromab: Phase 3 SAPPHIRE in SMA# The Phase 3 SAPPHIRE trial represents the most advanced clinical success for any myostatin-targeted therapy: 188 patients aged 2-21 with Type 2 or 3 SMA, all receiving background SMN-targeted treatment Primary endpoint met : Clinically meaningful improvement on HFMSE (Hammersmith Functional Motor Scale Expanded) versus placebo (p=0.0192), with a mean difference of 1.8 points Functional responders : 30.4% of apitegromab patients achieved 3 or more point HFMSE improvement versus 12.5% placebo Upper limb function : Consistent improvement measured by RULM (Revised Upper Limb Module) Published in The Lancet Neurology This is notable because it overcomes the historical "mass-function disconnect" that plagued earlier myostatin inhibitors -- apitegromab improved actual motor function, not just muscle mass
The implications for longevity and healthspan are substantial, as preventing type 2 diabetes eliminates a cascade of downstream complications that would otherwise accumulate over decades
ONeil PM, et al
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