Author Archives: Charles Moore

Biotech Focuses on Gene Therapy for Huntington’s, Heart Disease, Hemophilia B

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…

Discovery Shows Enzyme May Protect Against Effects of Degenerative Brain Diseases

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…

Huntington’s Gene-inducing Damage Might Have a Control Mechanism

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…

Neurinox Scientists Find Oxygen Radicals Play Dual Role in Neurodegenerative Diseases

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…

FDA Seeks Input on Huntington’s, Parkinson’s Disease Impact, Treatment

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…

MitoAction hosts 4th Mitochondrial Disease Clinical Conference

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…