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February 14, 2020February 14, 2020

Gene Discovery in Fruit Fly Study May Help Uncover Treatments for Mitochondrial Disease in People

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Scientists have discovered that the tam gene can be targeted to reverse the symptoms of harmful mutations in mitochondrial genes in a fruit fly study, a finding that may ... Read more

February 12, 2020February 12, 2020

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

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The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous ... Read more

February 7, 2020February 7, 2020

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

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The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over ... Read more

January 31, 2020January 31, 2020

Khondrion Doses First Patient in Phase 2b Trial Testing Sonlicromanol for Mitochondrial Diseases

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Khondrion has dosed the first patient in a Phase 2b clinical trial evaluating sonlicromanol, its lead investigational therapy for treating a range of mitochondrial diseases in adults. Sonlicromanol is ... Read more

January 30, 2020February 21, 2020

Nonprofits RARE Courage, All Wheels Up Partner on Survey Seeking to Improve Air Travel for Wheelchair Users

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Kevin Schaefer hadn’t been in an airport since he was 4 years old, so he had been looking forward to flying from his home in Cary, North Carolina, to ... Read more

January 24, 2020January 24, 2020

Entrada Developing Enzyme Replacement Therapy ENTR-501 to Treat MNGIE Disease

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Entrada Therapeutics is developing an enzyme replacement therapy, called ENTR-501, as a potential new treatment strategy for people with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). MNGIE is a rare inherited disease caused ... Read more

January 17, 2020January 17, 2020

NeuroVive Soon to Recruit Patients With Primary Mitochondrial Disease to Test its Oral Treatment Candidate

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NeuroVive Pharmaceutical AB has completed recruitment of healthy subjects for the second part of an ongoing Phase 1a/b study testing its oral treatment candidate KL1333 for primary mitochondrial disease. ... Read more

January 10, 2020January 10, 2020

Elamipretide Failed to Meet Promise of Earlier Trial Results for Primary Mitochondrial Myopathy, Data Show

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Treatment candidate elamipretide did not meet expectations stemming from promising early trial results in patients with primary mitochondrial myopathy (PMM), data from a Phase 3 trial show. Elamipretide is ... Read more

January 3, 2020January 3, 2020

New Fluorescent Marker Allows Detailed Study of Mitochondria in Live Cells, Study Reports

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MitoPB Yellow, a new fluorescent specific marker, allows scientists to study live mitochondria, possibly leading to a better understanding of mitochondrial diseases and to new treatments, researchers in Japan ... Read more

December 20, 2019December 20, 2019

LHON Natural History Study Highlights Improvements in Visual Acuity From Lumevoq Treatment

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Patients with Leber hereditary optic neuropathy (LHON), a rare genetic mitochondrial disease that causes vision loss, experienced a dramatic and mostly irreversible loss of visual acuity, according to an ... Read more