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…
AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…
The Cleveland Team Hope Walk & 5K Run is on tap for Aug. 15 to raise funds to help the Huntington’s Disease Society of America (HDSA) Northeast Ohio Chapter to improve the lives of Huntington’s disease (HD) patients and their families. The event, which seeks to raise $36,000…
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…
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