Author Archives: Charles Moore

Gene therapy biotech uniQure is prioritizing its drug pipeline to develop treatments for Huntington’s disease, hemophilia B, and its partnered gene therapy programs associated with uniQure’s collaboration with Bristol-Myers Squibb (BMS) in cardiovascular disease. The company will restructure its research and development organization in the Netherlands and consolidate manufacturing in…

A multi-institutional study led by a team of biomedical researchers in the lab of Hui-Chen Lu at Indiana University at Bloomington, Indiana, has discovered evidence indicating that the enzyme nicotinamide mononucleotide adenylyl transferase 2 (NMNAT2) may have protective qualities against debilitating effects of certain degenerative brain diseases called proteinopathies, that…

Cambridge, England-based light therapy specialist Lumie will supply 24 of its most powerful light boxes, called Lumie Brazil, for use in a Huntington’s disease research study at the Cambridge School of Clinical Medicine’s Neurology Unit. The Lumies are being donated to…

Texas Tech University Health Sciences Center (TTUHSC) researchers have identified a molecule — mitochondrial division inhibitor 1 (Mdivi1) — believed to hold promise as a potential treatment for Huntington’s disease. Mitochondria are specialized organelles in a cell responsible for creating the energy needed by the body to sustain life. An…

Neurodegenerative diseases (ND) represent a major and growing healthcare problem, especially in developed nations with aging populations. These disorders affect mostly adults and can last for decades, causing long-term suffering to patients and their families. Neurodegeneration also contributes to the development of chronic debilitating diseases affecting younger people, such as…

In 2013, the U.S. Food and Drug administration (FDA) selected Huntington’s disease (HD) and Parkinson’s disease (PD) as the focus of a three-year public consultation project for Patient-Focused Drug Development. This initiative involves working toward a better understanding of patient perspectives on the severity of these diseases and evaluation of…

Huntington’s disease is among a select group of disorders for which eligible patients can be prescribed medical marijuana in New York State. A bill signed this week by N.Y. Gov. Andrew M. Cuomo, passed by both houses of the legislature in June with overwhelming bipartisan support, expands a 2014 state law establishing medical marijuana access by…

Mitochondria, the true energy power plants of cellular activity, play a vital role in energy production and in several other essential cellular processes, including lipid and amino acid metabolism, the urea cycle, iron sulfur cluster assembly, iron homeostasis and cell death. In their normal function, mitochondria are able to release…

As part of its ongoing effort to improve mitochondrial disease patient care, MitoAction — a nonprofit organization dedicated to improving the quality of life for children, adults, and families living with mitochondrial disease through support, education, outreach, advocacy, and clinical research initiatives — in collaboration with the organization’s Medical Advisory…

A new international study by scientists at the University of Granada (UGR) in Spain, the University of Wisconsin-Madison, Columbia University in New York, and Rutgers, The State University of New Jersey, has uncovered new data on the coenzyme Q10 (CoQ10), a molecule which is normally synthesized within an organism’s own…