Parkinson’s disease patients experience reduction in symptoms with non-surgical ultrasound treatment

Patients with Parkinson’s disease achieved a significant improvement in their tremors, mobility, and other physical symptoms after having a minimally-invasive procedure involving focused ultrasound, according to a new study published in the New England Journal of Medicine.

The study, conducted by researchers at the University of Maryland School of Medicine (UMSOM), has led to FDA approval of a focused ultrasound device that provides a novel treatment option without requiring an incision. “Focused ultrasound was really transformative. So many of my fine motor skills have returned. I’m putting on eyeliner again and taking showers again without falling,” said Melanie Carlson who participated in the study. Click here to learn more.

FDA approves omaveloxolone (SKYCLARYS) as first Friedreich’s ataxia drug

The FDA has approved omaveloxolone (SKYCLARYS), an agent developed by Reata Pharmaceuticals for the treatment of Friedreich ataxia in adults and adolescents aged 16 years and older, making it the first and only therapy approved for the indication.

According to Susan Perlman, MD, clinical professor of neurology at the David Geffen School of Medicine of UCLA, “The approval of SKYCLARYS represents an important step forward in the treatment of Friedreich's ataxia, providing physicians with the first disease-specific treatment option approved for patients living with this ultra-rare and progressive disease.” Click here to learn more.

Praxis share price halved after essential tremor fail, but biotech still plans phase 3

Praxis Precision Medicines isn’t letting a midphase fail stand in the way of its plans in essential tremor. While its selective T-type calcium channel blocker failed to beat placebo on the primary endpoint, Praxis zeroed in on secondary measures and post hoc analyses to make the case for further development. 

The candidate, ulixacaltamide, is designed to improve the symptoms of essential tremor by normalizing burst firing in a sensory-motor network that is implicated in the disorder. To test that idea, investigators randomized 132 people with essential tremor to receive placebo or one of two regimens of ulixacaltamide. Click here to learn more.

AI system identifies new drug candidates for Parkinson’s disease

A new study by the University of Toronto suggests that the language used by researchers in describing their results can be utilized to uncover new treatments for Parkinson’s disease. The study utilized AI to find an existing anti-cholesterol medication that has the capability to enhance the disposal of mitochondria, which are cellular components responsible for energy production and are affected in Parkinson’s disease. Click here to learn more.

National study seeks patients with ATP1A3 mutations

ATP1A3 disorders are the subject of a five-year, $3.3 million National Institutes of Health-funded clinical study on the Clinical Genetic and Cellular Consequences of Mutations in ATP1A3. The multi-institutional team is led by Allison Brashear, MD, vice president for health sciences at the University at Buffalo, dean of the Jacobs School of Medicine and Biomedical Sciences at UB and professor in the Department of Neurology.

"We are actively recruiting patients with ATP1A3 disorders for our study," says Brashear. "The goal is to generate a comprehensive natural history of the symptoms and progression of ATP1A3 disorders, which put such a heavy and sudden burden on patients. The immediate hope is to expand what we know about these disorders to begin working toward new treatments and ultimately improving the quality of patients’ lives." Click here for more information on this clinical study and how to enroll.