Levels of proteins derived from prodynorphin (PDYN) — ultimately used during cell-to-cell signaling in the brain — are decreased in the fluid surrounding the brains of people with Huntington’s disease (HD), a new small study suggests. These findings indicate that PDYN-derived proteins could be used as specific biological…
The first U.S. patients have been enrolled in PROOF-HD, a Phase 3 clinical trial investigating pridopidine as a treatment for Huntington’s disease, according to developers Prilenia Therapeutics. PROOF-HD (NCT04556656) will enroll up to 480 people with early-stage Huntington’s, and is currently recruiting at more than 60…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is…
The Hereditary Disease Foundation‘s (HDF) has announced the first winner of its Nancy S. Wexler Young Investigator Prize, supporting excellence and creative thinking in Huntington’s disease research. The inaugural award went to Osama Al-Dalahmah, a neuropathology instructor in the department of pathology and cell biology…
A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…
The U.S. Food and Drug Administration (FDA) has given orphan drug designation to the oral therapy branaplam (LMI070) as a potential treatment for Huntington’s disease, the therapy’s developer, Novartis, announced. The designation is given to medications that have the potential to treat rare diseases — defined in…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
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…
No significant safety concerns associated with AMT-130, uniQure’s experimental gene therapy for Huntington’s disease, were identified in the first two patients participating in a Phase 1/2 trial assessing the treatment’s safety, tolerability, and efficacy. These were the main conclusions that came out of the first meeting of an…
Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…
Get regular updates to your inbox.