Arimoclomol in infants with Niemann-Pick disease type C: Results from the phase 2/3 open-label pediatric substudy
Eugen Mengel, Laila Arash-Kaps, Stephanie Grunewald, Sabine Weller Grønborg, Natalie Berger, Hadeel Shammas, Christine í Dali · 2026
Read Arimoclomol in infants with Niemann-Pick disease type C: Results from the phase 2/3 open-label pediatric substudy by Eugen Mengel, Laila Arash-Kaps, Stephanie Grunewald, Sabine Weller Grønborg, Natalie Berger, Hadeel Shammas, Christine í Dali (2026) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/PMC13310637.txt.
Description
Eugen Mengel and co-authors report a trial substudy of a drug for a rare lipid storage disease in infants in a 2026 article. Niemann-Pick disease type C harms the brain and organs from early life, and arimoclomol is approved in the United States from age two, paired with miglustat. Safety and handling in babies under two were untested.
Five infants aged 14 to 23 months at screening received arimoclomol beside standard care for up to 36 months, all also taking miglustat. Safety led the outcome list, with development scores, biomarkers, and drug levels behind. Most of the 108 reported side events were mild or moderate, though 15 serious events fell in two patients. Exposure ranged widely, from 72 to 1109 days across the small group.
Catalog record
| CREATOR | Eugen Mengel, Laila Arash-Kaps, Stephanie Grunewald, Sabine Weller Grønborg, Natalie Berger, Hadeel Shammas, Christine í Dali |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 45183 |
| LICENSE | https://creativecommons.org/licenses/by/4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |