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Brain-derived neurotrophic factor (BDNF) may not be the biomarker for Huntington’s disease that it was previously thought to be, a recent study reported. The progressive loss of BDNF, a factor that helps neurons grow and survive, associates with Huntington’s progression in mouse disease models, and past studies found it at reduced…
A history of high alcohol intake seems to be linked to increased motor impairment and a higher burden of psychiatric symptoms in Huntington’s disease patients, a large international study suggests. The observational study, “Genetic and Environmental Contributors to Neurodegeneration: An Exploration of the Effects of Alcohol…
At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…
Treatment with pridopidine rescues the function of mitochondria — the cell’s powerhouses — boosting energy production, and reduces oxidative stress in models of Huntington’s disease, preclinical research shows. According to researchers, these findings support the therapeutic potential of pridopidine and its ongoing clinical development. Pridopidine is a small oral…
To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…
The first two patients have been enrolled in the high-dose group of a U.S. clinical trial testing the safety and efficacy of AMT-130 as a potential gene therapy for Huntington’s disease. “We are very pleased with the progress that we continue to make in this Phase 1/2 clinical trial and…
Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…
Galyan Bio plans to initiate a clinical trial of a small molecules candidate that aims to slow the progression of Huntington’s disease next year, working in with Bicoll GmbH. Galyan Bio is developing and optimizing small molecules as oral treatments of Huntington’s and other neurodegenerative disorders, which work by targeting disease-causing mutant…
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…
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