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July 20, 2018July 20, 2018

HotSpot Therapeutics Secures $45M to Advance Drug Discovery Platform

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HotSpot Therapeutics has secured $45 million to advance its drug discovery strategy that identifies so-called “hot spots” in proteins, allowing for their precise targeted regulation. Using this technology, HotSpot Therapeutics ... Read more

July 13, 2018July 13, 2018

Cerebrospinal Fluid Biomarker NfL Can Help Monitor Nerve Cell Degeneration, Study Says

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Analysis of a cerebrospinal fluid protein called neurofilament light (NfL), which is linked to nerve cell damage, can help monitor the neurodegenerative process in patients with mitochondrial diseases. This ... Read more

July 6, 2018July 6, 2018

NeuroVive Licenses BridgeBio Subsidiary to Develop LHON Therapies

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BridgeBio Pharma has entered into an exclusive licensing agreement with NeuroVive Pharmaceutical AB for the development of a subset of chemical compounds under NeuroVive’s NVP015 program. The goal is to further ... Read more

June 29, 2018June 29, 2018

Older Moms More Likely to Pass Along Mitochondrial DNA with Mutations, Study Finds

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A mathematical model that can predict the nature of the mitochondrial DNA a child will inherit from its mother was part of a study that also found that maternal ... Read more

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

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