| abstract |
The present invention relates to the use of 0-(3-piperidino-2-hydroxy-l-propyl)- nicotinic amidoxime (BGP15), its tautomers, enantiomers and pharmaceutically acceptable salts thereof the treatment of diseases characterised at least in part by an excessive mitochondrial fragmentation activity or by a reduced mitochondrial fusion activity such as pulmonary fibrosis, age related macular degeneration, retinitis pigmentosa, heart failure with preserved ejection fraction, an inherited mtDNA depletion disorder (such as Alper's disease) or an inherited mtDNA deletion/mutation disorder (such as progressive external ophthalmoplegia, an ataxia-neuropathy spectrum disorder or mitochondrial neurogastrointestinal encephalomyopathy). |