Author Archives: Margarida Azevedo, MSc

Mitochondrial disease is very costly to American patients and better therapies are needed urgently, say researchers at the Children’s Hospital of Philadelphia (CHOP). Their National Institutes of Health-funded study, “Hospitalizations for mitochondrial disease across the lifespan in the U.S.,” appeared in the April 2017 issue of Molecular Genetics…

NeuroVive Pharmaceutical has received a Swedish innovation agency grant worth $118,000 that it will use to accelerate work on a treatment it is developing for genetic mitochondrial disease. The agency, Vinnova, made the 1-million-kroner grant under the government’s Swelife call program, which supports collaborations between the medical system, universities and industry that…

KL1333, a new potential treatment for orphan genetic mitochondrial disorders, is entering Phase 1 studies, thanks to an accord between NeuroVive and Yungjin Pharm. Under terms of the contract, Sweden’s NeuroVive gains exclusive worldwide rights to develop and commercialize KL1333 except in South Korea and Japan. The deal could generate up to…

Santhera Pharmaceuticals, developer of the mitochondrial eye disease therapy Raxone, is taking a seat on the United Mitochondrial Disease Foundation industry advisory council. Dr. Jodi Wolff, who is known for her expertise on the disease and her patient advocacy work, will represent Santhera on the council. She is the company’s director…

GenSight Biologics will present data from studies of its gene therapy GS010 for Leber hereditary optic neuropathy (LHON), a mitochondrial disease, at an upcoming Neuro-Ophthalmology Society conference. According to a press release, the one oral and two poster presentations will be given at the 2017 North American Neuro-Ophthalmology Society‘s (NANOS)…

If you or a family member has mitochondrial disease, you might want to attend the upcoming 2017 Mitochondrial Medicine Southeast Regional Symposium, organized by the United Mitochondrial Disease Foundation (UMDF), the Foundation for Mitochondrial Medicine (FMM) and the University of Alabama Birmingham (UAB). The symposium will…

GenSight Biologics has completed enrolling patients in its Phase 3 REVERSE clinical trial evaluating GS010 as a treatment for Leber’s Hereditary Optic Neuropathy (LHON). LHON is a rare inherited mitochondrial disease that is passed on by the mother. It causes degeneration of retinal cells in an offspring, leading to…

NeuroVive will collaborate with the Children’s Hospital of Philadelphia (CHOP) and mitochondrial expert Marni J. Falk to evaluate experimental compounds the company is developing for the disease. NeuroVive is working on substances that are both delivery vehicles and treatments. The delivery-vehicle components get the treatments to the cells, where they activate…

The National Science Foundation (NSF) awarded a Penn State Brandywine assistant professor of biology, Megan Povelones, a National Science Faculty Early Career Development (CAREER) grant to support her study of mitochondrial structure and function. The five-year grant is one of the NSF’s most-sought early career development honors, given to teacher-researchers working…

The United Kingdom is the first country to approve “three-parent” fertility treatments after receiving a nod from regulatory authorities, a decision that is likely to allow women in the UK with mitochondrial disease to have a healthy baby. Lawmakers in the UK had voted earlier this year to allow the…