Enzyme Replacement Therapy for Lysosomal Storage Diseases
Автор: March Community
Загружено: 2025-10-04
Просмотров: 26
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For families navigating Lysosomal Storage Diseases, the routine of Enzyme Replacement Therapy is a familiar path. But what if the future holds new and different options? In this episode of March Forward, we're exploring the cutting-edge science that could change how we treat these complex conditions.
We're asking the big questions that many in our community think about:
🧠 Can we get treatments safely past the blood-brain barrier?
🤔 How do you weigh a proven, lifelong therapy against a one-time treatment with unknown long-term effects?
🔬 Are we getting closer to teaching the body to accept therapy, without harsh drugs?
💊 What will it take to finally develop a simple pill for these complex diseases?
Join us as we break down the science, explore the potential of "Trojan horse" therapies, gene therapy, and more, all with the goal of empowering our community with knowledge. We're in this together. ❤️
#LysosomalStorageDisease #EnzymeReplacementTherapy #RareDisease #PatientCommunity #ERT #GeneticDisorders #MarchForward
March Forward - Podcast by March.ai
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