News

Presymptomatic Disease Changes One’s View of Time, Patients Say

Huntington’s disease patients who have not yet developed symptoms have a different relationship with time than otherwise-healthy individuals, according to a new study based on patient interviews. The findings highlight a need for long-term psychological support for people with presymptomatic Huntington’s to help them develop proactive coping strategies and…

Glia Brain Cells May Open Door to Treatment Strategy

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…

Register Now for Global Genes’ RARE Patient Advocacy Summit

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…

Hearing on Huntington’s Appreciated, But Parity Act Is Priority

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…

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

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…

VO659 Earns Orphan Drug Designation

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…

Advocates Lobby US Congress During Virtual Rare Disease Week

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…

US Patient Groups Give Thumbs-Up to Rule Against Surprise Billing

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…