News

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…

Brain cells called glia work to protect the brain from damage in Huntington’s disease by reducing the expression of genes involved in maintaining connections between nerve cells, a new study indicates. The findings suggest that targeting glia could be a useful strategy for Huntington’s treatment, and also highlight that…

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…

While the Huntington’s Disease Society of America (HDSA) is pleased a U.S. House of Representatives subcommittee recently held a key hearing that featured Huntington’s disease, it continues to call for passage of bipartisan parity legislation. The House Energy and Commerce Subcommittee on Health hearing was focused at better…

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Vico Therapeutics’ VO659, an investigational antisense oligonucleotide gene-silencing therapy for Huntington’s disease. “We are delighted that FDA has granted this orphan-drug designation,” Rupert Sandbrink, MD, PhD, chief medical officer of VICO, said in a…

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…

People with Huntington’s disease who have had more formal education tend to be diagnosed earlier and have milder symptoms than those with less formal education, a new study indicates. The study, “The effect of education on symptom onset and severity of Huntington’s disease,” was published in …

The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…

Progression in Huntington’s disease may be due more to a gradual loss of the system that maintains the health of brain cells than to accumulated damage caused by the disease itself, a computational analysis of large and complex datasets suggests. This analysis, called Geomic, showed that while brain cells most vulnerable…