uniQure recently showed that its gene therapy candidate AMT-130 improves motor coordination and survival in mouse models of Huntington’s disease (HD). The preclinical study results were presented at the European Society of Gene and Cell Therapy (ESGCT) 25th Anniversary Congress, held Oct. 17-20 in Berlin, Germany. The…
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More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…
Researchers have come up with a new method to study the interactions between cell components that are thought to be defective in patients with Huntington’s disease and other neurodegenerative conditions. But while the study answered some initial questions of how these cell components, or organelles, collaborate, much work remains,…
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#NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says
As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…
Three Huntington’s disease (HD) patient advocacy organizations in Europe and North America recently launched a collaborative effort to give patients and the HD community more of a voice in clinical research. The newly formed Huntington’s Disease Coalition for Patient Engagement (HD-COPE) aims to give Huntington’s patients a platform to express their…
The U.S. Food and Drug Administration (FDA) has awarded orphan drug status to uniQure’s proprietary candidate for Huntington’s disease (HD), a first in the history of Huntington’s treatment. No approved therapies exist to target the underlying cause of Huntington’s, a disease caused by a mutation in the huntingtin gene .
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Measuring PFS Outcome Might Help Researchers Evaluate Huntington’s Progression, Study Suggests
Identifying Huntington’s disease progression can be a challenge in clinical trials. Now, an international team of researchers proposes in an article published in JAMA Neurology the use of a new composite measure to help evaluate disease progression prior to a motor diagnosis. Progression of Huntington’s disease is characterized by the…
The National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH) has awarded a grant of more than $2.5 million to support the development of HDWear, a telecare technology that uses wearable sensors to remotely monitor motor function in Huntington’s disease (HD) patients.
Less is known about the late stages of Huntington’s disease than about its earlier stages, particularly concerning patients enrolled in hospice care. Researchers studying this patient group recently concluded that they would benefit from more extensive involvement of hospice in end-of-life care. The study, “Utilization of hospice services in…
GeneFo will offer a free webinar on how adding humor to Huntington’s patients’ lives can help improve their symptom management, emotional wellbeing, ability to cope with adversity, and social and work interaction. The webinar, which is aimed at caregivers as well patients, will start at 1 p.m. U.S. Eastern…
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