Lindsey Shapiro

2 days ago | friedreichsataxianews.com | Lindsey Shapiro

Using MRI scans, a team of researchers identified several differences in the brain and spinal cord of people with Friedreich’s ataxia (FA) versus those without the rare genetic condition, which have the potential to serve as biomarkers of disease progression in clinical trials. The findings come from TRACK-FA (NCT04349514), a large, international natural history study that’s monitoring long-term neuroimaging changes among people with and without FA.

3 days ago | alzheimersnewstoday.com | Lindsey Shapiro

Leqembi (lecanemab) has won marketing authorization in the European Union for the treatment of certain adults with early Alzheimer’s disease, making it the first therapy that targets an underlying cause of the neurodegenerative condition to be approved in the region. The treatment is indicated for adults with mild cognitive impairment or mild dementia due to Alzheimer’s who have confirmed buildup in the brain of the amyloid-beta protein — the disease hallmark that Leqembi targets.

3 days ago | gaucherdiseasenews.com | Lindsey Shapiro

Treatment with Cerdelga (eliglustat), a substrate reduction therapy approved in the U.S. for adults with type 1 Gaucher disease, was safe and showed therapeutic potential in children with the condition, a real-world study finds. “This case series highlights real-world experience with [Cerdelga] in pediatric [Gaucher type 1] patients, providing important insights into its potential to improve clinical outcomes and optimize disease management,” the study’s researchers wrote.

3 days ago | pulmonaryhypertensionnews.com | Lindsey Shapiro

On May 5, more than 80 organizations around the world will join to raise awareness of pulmonary hypertension (PH), a serious but often under-recognized cardiovascular disease affecting more than 75 million people worldwide. World Pulmonary Hypertension Day, an annual event spearheaded by PHAEurope, also aims to promote healthcare and treatment access for people with PH and support research efforts toward a cure.

6 days ago | parkinsonsnewstoday.com | Lindsey Shapiro

Scientists have developed a blood test to detect Parkinson’s disease in its very early stages, before a person experiences the first symptoms that would normally prompt a Parkinson’s diagnosis, a study showed. After identifying that people with Parkinson’s have alterations in two types of transfer RNA fragments (tRFs), molecules that play a variety of roles in cellular function, researchers developed a diagnostic test to measure the ratio between the molecules.