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PUBLICATIONS AND PRESENTATIONS
Poster Presentations - All Dates
ACMG - 04/08/2026
Analysis of NPC1 Genotypes: Findings From the US Arimoclomol Expanded Access Program for Niemann-Pick Disease Type C
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22nd Annual WORLDSymposium - 02/03/2026
Efficacy of Arimoclomol Combined With Miglustat at Months 3, 6, 9 and 12 of the Double-Blind, Randomized, Placebo-Controlled NPC002 Trial
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22nd Annual WORLDSymposium - 02/03/2026
Multi-year subgroup analyses of Niemann-Pick disease type C participantstreated with arimoclomol in the US early access program (EAP)
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22nd Annual WORLDSymposium - 02/03/2026
Real-world safety and effectiveness arimoclomol in patients with NPC:outcomes from the US early access program (EAP) over a 4-year period
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22nd Annual WORLDSymposium - 02/03/2026
Long-term safety and effectiveness of arimoclomol adult and pediatricNiemann-Pick disease type C patients in the US early access program (EAP)
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Poster Presentations - 2026
ACMG - 04/08/2026
Analysis of NPC1 Genotypes: Findings From the US Arimoclomol Expanded Access Program for Niemann-Pick Disease Type C
Read More
22nd Annual WORLDSymposium - 02/03/2026
Long-term safety and effectiveness of arimoclomol adult and pediatricNiemann-Pick disease type C patients in the US early access program (EAP)
Read More
22nd Annual WORLDSymposium - 02/03/2026
Real-world safety and effectiveness arimoclomol in patients with NPC:outcomes from the US early access program (EAP) over a 4-year period
Read More
22nd Annual WORLDSymposium - 02/03/2026
Multi-year subgroup analyses of Niemann-Pick disease type C participantstreated with arimoclomol in the US early access program (EAP)
Read More
22nd Annual WORLDSymposium - 02/03/2026
Efficacy of Arimoclomol Combined With Miglustat at Months 3, 6, 9 and 12 of the Double-Blind, Randomized, Placebo-Controlled NPC002 Trial
Read More
Poster Presentations - 2025 to 2024
Poster Presentations - 2023 to 2022
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