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…
Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…
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…
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