Author Archives: Patricia Inacio PhD

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

A Phase 1/2 trial testing the safety and efficacy of AMT-130, uniQure’s experimental gene therapy for Huntington’s disease, can continue dosing after a positive recommendation from an independent board. The data safety monitoring…

uniQure has completed patient enrollment for the first group participating in a Phase 1/2 trial assessing the safety and efficacy of AMT-130, its experimental gene therapy for Huntington’s disease…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

Anima Biotech has partnered with Takeda Pharmaceuticals to develop small molecules that control mRNA translation — the process by which the information encoded in a person’s DNA is “translated” into a protein — as potential therapies for neurological disorders with a genetic cause, including Huntington’s disease. Huntington’s…

A Phase 1 trial will evaluate PTC Therapeutic’s PTC518, its investigational therapy for Huntington’s disease, in healthy volunteers, the company announced. The trial’s first results are expected in the first half of 2021. “The initiation of the clinical trial to evaluate PTC518 for the Huntington’s disease program is…

The U.S. Food and Drug Administration (FDA) is putting on hold Voyager Therapeutics’ request to test VY-HTT01, its investigational gene therapy for Huntington’s disease, in a clinical trial. The clinical hold will be kept in place until certain issues with VY-HTT01’s chemistry, manufacturing, and controls are…

The first patients have been enrolled in a clinical study of the natural history of Huntington’s disease (HD) and its biomarkers, to better inform how to measure and monitor early signs of the disease and support future clinical studies. The SHIELD HD study, sponsored and led by Triplet…

AFFiRiS’ C6-17, a candidate monoclonal antibody for the treatment of Huntington’s disease, is able to target and halt the spread of mutant huntingtin protein — the underlying cause of the neurodegenerative disorder — across cells, new preclinical data show. The study, “Inhibiting cellular uptake of mutant huntingtin using…