We read the recent study by Niemann et al. (2022) [...].
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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC9600176 | PMC |
http://dx.doi.org/10.3390/diagnostics12102343 | DOI Listing |
Drugs
January 2025
Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.
Arimoclomol (MIPLYFFA™), an oral small molecule that crosses the blood brain barrier and is thought to upregulate CLEAR (Coordinated Lysosomal Expression and Regulation) network genes and improve lysosomal function, is being developed by Zevra Therapeutics Inc., for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC). In September 2024, arimoclomol was approved for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older in the USA.
View Article and Find Full Text PDFEur Respir J
July 2024
Department of Infectious Diseases, Region Östergötland, Linköping University Hospital, Linköping, Sweden.
https://bit.ly/3wAVvFm
View Article and Find Full Text PDFClin Microbiol Infect
September 2024
Emerging Bacterial Pathogens Unit, Division of Immunology, Transplantation and Infectious Diseases, IRCCS San Raffaele Scientific Institute, Milan, Italy. Electronic address:
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