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Rare Disease Report®

The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.

The treatment could also be used for Alzheimer disease and mid-stage Huntington disease for patients who suffer from greater cognitive deficits. 

Pepinemab Yields Cognitive Benefit in Huntington Disease Patients

The RareWear program is designed to bridge medical device companies to rare disease patient applicants, seeking better disease and treatment monitoring.

Rare Genomics Institute Launches Free Rare Disease Device Program

New research suggests proposals aimed at reducing out of pocket costs may harm minimally-funded drug developers.

Medicare Part D Bills Could Harm Rare Disease Drug Development

An Investigational New Drug application was approved to clinically assess AMT-130 as the first potential drug marketed for the neurodegenerative disease.

FDA Clears Gene Therapy for Huntington Disease Assessment

An isotopic-reinforced polyunsaturated fatty acids shows a reduction in cognitive deficits in a preclinical in vivo model of Huntington disease.

D-PUFA Drug Candidate Shows Cognitive Improvement in Huntington Disease Patients

Results from the first multi-center interventional trial in premanifest HD research show that the antioxidant Coenzyme Q10 is associated with only mild or moderate side effects, even at higher doses. 

Coenzyme Q10 Well-tolerated in Patients with Huntington Disease Participating in a Multi-center Phase II Trial 

On the HCPLive Huntington disease page, resources on the topics of medical news and expert insight into HD can be found. Content includes articles, interviews, videos, podcasts, and breaking news on Huntington's chorea, and more.