Key Takeaway: The article discusses the presentation of real-world data collected from an expanded access program for Arimoclomol in Niemann-Pick Disease Type C. This presentation took place at the 52nd Child Neurology Society Annual Meeting on October 5, 2023. The focus on real-world data highlights the importance of practical insights in understanding treatment efficacy.
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POSITIVE FACTORS
Presentation at a reputable annual meeting.
Focus on real-world data collection.
Involvement of expanded access program.
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Full Press Release Details
< Medical Publications and Presentations
Real World Data Collection in Niemann-Pick Disease Type C – Data from Expanded Access Program with Arimoclomol
52nd Child Neurology Society Annual Meeting - 10/05/2023
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Frequently Asked Questions
What was presented at the Child Neurology Society Annual Meeting?
Real-world data from the expanded access program for Arimoclomol in Niemann-Pick Disease Type C.
When did the presentation take place?
The presentation occurred on October 5, 2023.
What is Arimoclomol used for?
Arimoclomol is being investigated for its efficacy in treating Niemann-Pick Disease Type C.
What is the significance of real-world data?
Real-world data provides practical insights into treatment efficacy and patient outcomes.