A Genetic Lottery
— Rebecca Field

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help…

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis…

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases…

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare…

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and…

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the…

Exercise and physical activity may benefit patients with Huntington’s disease (HD), a new review study shows. The study, “Physical Therapy and Exercise Interventions in Huntington’s Disease: A Mixed Methods Systematic Review,” appeared in the Journal of Huntington’s…

This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least 30 million Americans. The campaign kicks off Feb. 1 and…

Neurofeedback training may increase brain connectivity in patients with Huntington’s disease, possibly improving behavior and movement abnormalities, say British researchers at University College London. Their study, “Stimulating neural plasticity with real-time fMRI neurofeedback in Huntington’s disease:…

The European Medicines Agency (EMA) has granted orphan medicinal product designation to AMT-130, uniQure’s gene therapy candidate for the treatment of Huntington’s disease. AMT-130 is an experimental gene therapy that is being evaluated in studies designed…

uniQure expects to advance the clinical development of its Huntington’s disease investigational gene therapy, called AMT-130, during 2018. The company says it plans to file an investigational new drug application (IND) with the U.S. Food and Drug Administration (FDA)…

Keeping a strict eating schedule may help improve the quality of life of patients with Huntington’s disease and other neurodegenerative disorders, a mouse study suggests. The study, “Time restricted feeding improves circadian dysfunction as well as…