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June 22, 2018June 23, 2018

Stealth Raises $100M to Advance Elamipretide as Treatment for Mitochondrial Diseases

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Stealth BioTherapeutics announced that it has raised $100 million from investors to support the development of elamipretide and other compounds that might treat diseases involving mitochondria dysfunction. “We are gratified by ... Read more

June 15, 2018June 15, 2018

Mutations in Mitochondrial Gene ANT1 Linked to Risk of Bipolar Disease in Mouse Study

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Mutations in the mitochondrial gene ANT1 may increase the risk of bipolar disease by altering levels of serotonin transmission in the brain, a mouse study suggests. The study, “Ant1 mutant mice ... Read more

June 8, 2018June 8, 2018

Mutations in MRPS2 Gene Linked to Mitochondrial Dysfunction for First Time, Case Report Shows

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New genetic mutations affecting the MRPS2 gene were identified as the cause of mitochondrial disorder in two unrelated children, a case report shows. The study, “Bi-allelic Mutations in the ... Read more

June 4, 2018June 4, 2018

US ‘Right to Try’ Law Meets with Mix of Praise and Criticism, Including Among Those with Rare Diseases

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As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third ... Read more

June 1, 2018June 1, 2018

Surveys Details Reasons Mitochondrial Disease Patients Take Part – or Don’t – in Clinical Trials

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Two surveys of people with mitochondrial disease spotlighted factors that both motivate and hinder patients from taking part in clinical trials, and underscored the importance of working with patient ... Read more

May 29, 2018May 29, 2018

#ERDC2018 – When Treating Rare Disease Patients, Don’t Overlook Quality of Life, Panel Urges

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Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different ... Read more

May 25, 2018May 25, 2018

Phase 1 Clinical Trial Testing Potential Mitochondrial Disease Therapy KL1333 Shows Positive Results

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NeuroVive Pharmaceutical AB and Yungjin Pharm announced positive topline results from a Phase 1 clinical trial evaluating KL1333, an investigational treatment for genetic mitochondrial diseases. The randomized, single ascending dose trial ... Read more

May 18, 2018May 18, 2018

Mitochondrial Disease Treatment Candidate KH176 Shows Potential, Study Finds

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Khondrion’s mitochondrial disease treatment candidate KH176 has potential to be effective in a broad range of disorders, a new study reports. The research, “KH176 Safeguards Mitochondrial Diseased Cells from ... Read more

May 17, 2018May 17, 2018

‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35

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The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. ... Read more

May 17, 2018May 17, 2018

#ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say

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The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in ... Read more

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