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October 19, 2018October 19, 2018

NV354, Potential Therapy for Mitochondrial Diseases, Shows Promise in Preclinical Studies

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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 ... Read more

October 10, 2018October 10, 2018

Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

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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 ... Read more

September 21, 2018

#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

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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 ... Read more

September 20, 2018September 20, 2018

PISD is a Novel Mitochondrial Disease Gene, Case Report Shows

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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 ... Read more

September 14, 2018September 14, 2018

Elesclomol Can Help Restore Copper Levels in Patients with Mitochondrial Diseases, Researchers Find

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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 ... Read more

September 7, 2018September 7, 2018

$4M Grant Will Help Advance Research into NVP015 Treatment for Genetic Mitochondrial Disease

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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 ... Read more

August 31, 2018August 31, 2018

Investigational Therapy Jotrol Shows Potential to Treat Mitochondrial Disorders, Mouse Study Suggests

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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 ... Read more

August 24, 2018August 24, 2018

Short-Term Treatment With KH176 Lessens Depression, Improves Mood in Mitochondria Patients, Study Shows

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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, ... Read more

August 17, 2018August 17, 2018

Patients Groups Funding Research Project Focused on Developing New Nucleoside Therapy

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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 ... Read more

August 10, 2018August 10, 2018

Women with Mitochondrial Disease More Prone to Pregnancy Complications, Study Shows

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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, ... Read more

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