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NeuroVive Pharmaceutical’s therapeutic candidate NV354 for mitochondrial diseases, such as Leigh syndrome, was able to restore the body’s own energy substrate — succinate — in an in vivo model of mitochondria dysfunction, preclinical results show. Moreover, researchers observed that NV354 is efficiently delivered to the brain. Magnus Hansson, PhD, NeuroVive’s…

Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights…

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…

Mutations in the PISD gene can cause mitochondrial disease, a new study shows. The report, “PISD is a mitochondrial disease gene causing skeletal dysplasia, cataracts and white matter changes,” appeared in the journal bioRxiv. Classic mitochondrial disease is caused by dysfunctional energy production, and symptoms usually occur in organs that…

Elesclomol, an investigational anti-cancer drug, can help treat patients with mitochondrial diseases by restoring copper in the mitochondria, a new study shows. The study, “Elesclomol restores mitochondrial function in genetic models of copper deficiency,” was published in the journal Proceedings of the National Academy of Sciences. Mitochondria are…

The U.S. Department of Defense (DoD) has awarded a $4 million, three-year grant to the Children’s Hospital of Philadelphia (CHOP) to advance NeuroVive Pharmaceutical AB‘s NVP015 program for genetic mitochondrial diseases. The donation was made by DoD’s Office of the Congressionally Directed Medical Research Programs.

Jotrol, Jupiter Orphan Therapeutics’ lead therapeutic candidate, was found to markedly improve mitochondria production in the liver and brain of mice with Alzheimer’s disease, according to preclinical data. These recent results further demonstrate the potential of the investigational therapy to modify mitochondria stability and function, without major toxicity effects.

Short-term treatment with oral KH176 was safe and well-tolerated by mitochondria disease patients, and lessened their symptoms of depression while also having a positive effect in alertness and mood, a Phase 2 clinical trial shows. The study with those findings, “The KHENERGY study: Safety and efficacy of KH176 in…

The Mito Foundation and The Lily Foundation are joining efforts to support the development of a nucleoside therapy to treat mitochondrial depletion syndromes. This announcement from the two patients groups comes almost one year after the death of U.K. baby Charlie Gard due to a form of mitochondrial…

Women with mitochondrial disease or mitochondrial dysfunction are more susceptible to complications during pregnancy, and their newborns are more likely to have congenital defects, a retrospective study shows. The study, “Effects of mitochondrial disease/dysfunction on pregnancy: A retrospective study,” was published in Mitochondrion. Primary mitochondrial disease (MD) comprises several…