News

The Adira Foundation is inviting people with neurodegenerative diseases and their caregivers to join a grant proposal review committee. A nonprofit foundation, its mission is to unite people affected by some of most common neurodegenerative diseases — namely, Huntington’s disease, amyotrophic lateral sclerosis, multiple sclerosis, Alzheimer’s disease, and…

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…

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…

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…