Assembling the treatment puzzle in Niemann Pick C

What this episode covers

Dr Marc Patterson and Dr Eugen Mengel explain the challenges of treating Niemann Pick Type C. Recent studies have shown the efficacy of Miglustat and Arimoclomol but they may end up forming just part of the puzzle being built to manage this condition. Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment Eugen Mengel, et al https://doi.org/10.1002/jimd.12428 Long-term survival outcomes of patients with Niemann-Pick disease type C receiving miglustat treatment: A large retrospective observational study Marc C. Patterson, et al https://doi.org/10.1002/jimd.12245

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Frequently Asked Questions

How long is this episode of JIMD Podcasts?

This episode is 25 minutes long.

When was this JIMD Podcasts episode published?

This episode was published on October 22, 2021.

What is this episode about?

Dr Marc Patterson and Dr Eugen Mengel explain the challenges of treating Niemann Pick Type C. Recent studies have shown the efficacy of Miglustat and Arimoclomol but they may end up forming just part of the puzzle being built to manage this...

Can I download this JIMD Podcasts episode?

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