Study uncovers mechanism behind loss of smell in Parkinson's patients

About 75%–90% of Parkinson's patients report diminishing sense of smell, even prior to the onset of motor symptoms. In a new paper published in The Journal of Neuroscience, researchers at the Yale School of Medicine have determined the biological mechanisms behind this olfactory impairment by using an alpha-synuclein A30P mouse model used to mimic symptoms of Parkinson's in mice. Click here to learn more.

Gene editing for Huntington’s disease shows promise in pigs

Although researchers have know since 1993 that Huntington’s disease (HD) is caused by a repeat expansion in the huntingtin gene, figuring out how to correct this defect has not been easy. Recently, however, researchers at the Chinese Academy of Sciences in Guangzhou made a case for correcting the gene via CRISPR editing.

In a pig model of HD, a single brain injection of a viral vector carrying the gene-editing molecules cut the amount of mutant protein in half and improved the animal’s gait months later. Click here to learn more.

What the FDA approval of Skyclarys really means for the FA community

According to Movement Disorders Foundation Advisory Board member Matthew Lafleur, approval of the first treatment for Friedreich's ataxia changed nothing and everything. "While Skyclarys has been clinically proven to slow the rate of disease progression," writes Lafluer, a frequent contributor to the Friedreich's Ataxia News, "FA will continue to exist and have devastating outcomes. It’s tempting to hope that this drug will obliterate every challenge FA patients like me face, but that false expectation will only lead to disappointment." Click here to learn more.

Become a brain donor

Studies that have made important contributions to dystonia would not have been possible if volunteers had not decided in advance to donate their brains to research. Brain donation provides the opportunity to contribute to the field of dystonia research for future generations.

The Dystonia Medical Research Foundation works in partnership with the Harvard Brain Tissue Resource Center at McLean Hospital in Belmont, Massachusetts to assist people interested in registering as brain donors. Click here to learn more.

First healthy volunteer dosed in study of HER-096 for Parkinson’s

Herantis Pharma announced that the first healthy volunteer has been dosed in a clinical study that tests the safety and efficacy of its HER-096 treatment candidate for Parkinson’s disease. The Phase 1a clinical trial also is evaluating the ability of HER-096 to penetrate the blood-brain barrier — a biological roadblock that prevents bacteria and parasites in the bloodstream from getting into the brain, but which also stops medications from doing so. Top-line data on the drug's safety and tolerability is expected by year's end. Click here to learn more.