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http://dx.doi.org/10.1038/s41380-022-01781-7 | DOI Listing |
Eur Respir J
December 2024
Division of Pulmonary and Critical Care Medicine, Department of Internal Medicine, University of Michigan Medical School, Ann Arbor, MI, USA
J Cachexia Sarcopenia Muscle
February 2025
School of Traditional Chinese Medicine, Southern Medical University, Guangzhou, China.
Drugs
December 2024
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 PDFJ Antimicrob Chemother
December 2024
Department of Microbiology and Instituto Biosanitario de Granada, University Hospital of Virgen de las Nieves, Avda. Fuerzas Armadas s/n, 18014 Granada, Spain.
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