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Lessons learned at the Gene Therapy for Rare Disorders Summit, especially insights for Natural History Studies.
To the patients who benefit from treatments that may have otherwise never been developed, your contribution would mean everything.
Estimating the cost of manufacturing an AAV Gene Therapy by comparing industry benchmarks and published doses used in clinical…
An exploration of value based pricing, how willingness-to-pay and ´novel concepts are inflating the cost effectiveness of gene therapies.
Crowdfunding is a growing democratic alternative to venture capital. Can it also provide the fuel to accelerate novel therapies…
Economic challenges in rare disease gene therapy, intensified by Eroom's law, demand innovative solutions. Amid accusations of pharmaceutical oligopoly…