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December 14, 2018December 14, 2018

GS010 Improves Visual Acuity, Quality of Life in LHON Patients, Phase 3 Trial Shows

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GS010, a gene therapy developed by GenSight Biologics, improves visual acuity and quality of life in patients with Leber hereditary optic neuropathy (LHON), a rare genetic mitochondrial disease that causes ... Read more

December 7, 2018December 7, 2018

NeuroVive Secures Funding to Advance NV354 for Potential Mitochondrial Disease Treatment

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Swedish innovation agency Vinnova has awarded $166,252 (1.5 million kroner) to NeuroVive Pharmaceuticals for the preclinical development and large-scale manufacture of its therapeutic candidate NV354 for mitochondrial diseases. The funds ... Read more

November 30, 2018November 30, 2018

Mitochondrial DNA from Fathers Could Lead to New Therapies, Study Suggests

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A new study found rare cases of mitochondrial DNA (mtDNA) being passed down from fathers in three unrelated families. The surprising findings could lead to novel treatments for inherited ... Read more

November 16, 2018November 16, 2018

Correcting Precise RNA Alterations May Restore Protein Production in MERRF, Study Suggests

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Restoring a missing transfer RNA (tRNA) modification improved protein production and stability in mitochondria — the cell’s power plants — in a patient with myoclonic epilepsy with ragged red fibers ... Read more

November 9, 2018November 9, 2018

Mount Sinai’s Icahn School of Medicine Cited for Commitment to Mitochondrial Care

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Owing to its commitment to mitochondrial medicine, the Icahn School of Medicine at Mount Sinai has been designated a certified Mitochondrial Medicine Center by the Mitochondrial Care Network (MCN). ... Read more

November 2, 2018November 2, 2018

New Mutations in NDUFA6 Gene Found in Four Cases of Mitochondrial Dysfunction

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New mutations affecting the NDUFA6 gene were identified as the cause of early-onset severe mitochondrial disorder in four unrelated children, a report shows. The study, “Bi-allelic Mutations in NDUFA6 ... Read more

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

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