A therapy for Huntington’s disease called INT41 was granted orphan drug designation by the U.S. Food and Drug Administration (FDA), bringing the treatment a step closer to being tested in a clinical trial. The announcement was made by Vybion Inc., a…
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Taking care of parents with Huntington’s disease evokes thoughts of “this could be me” in young caregivers and affects their planning for predictive genetic testing, a study shows. The study, ““This could be me”: exploring the impact of genetic risk for Huntington’s disease young caregivers,” was published in…
A bioactive compound present in raspberries called salidroside can prevent the cellular toxicity that occurs as a consequence of protein accumulation in certain neurodegenerative diseases, including Huntington’s disease, a study reports. The study, “Identification and microbial production of the raspberry phenol salidroside that is active against Huntington’s disease,” was published…
A deficiency in energy metabolism in the cells of patients with Huntington’s disease can partly explain the variation in age of onset among individuals, a study shows. The study, “Bioenergetics in fibroblasts of patients with Huntington disease are associated with age at onset,” was published in the journal…
The juvenile form of Huntington’s disease, which affects patients younger than 20, is particularly aggressive and associated with an early onset of symptoms, a retrospective study has found. Based on the study’s findings, researchers have also suggested that a revision of the current classification of juvenile Huntington’s disease may…
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#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach
With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit and affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate,…
Suppressing the expression of the defective huntingtin gene using antisense oligonucleotides lowered anxiety-like behavior in a mouse model of Huntington’s disease, a study reports. These compounds were also efficiently delivered into the brains of nonhuman primates and targeted specific areas involved in cognition and psychiatric function. This study, “…
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Gene Therapy VY-HTT01 Can Protect Brain Cells from Damage Caused by HTT Faulty Gene, Results Show
One-time delivery of Voyager Therapeutics’ lead candidate gene therapy, VY-HTT01, effectively reduced the levels of the faulty gene responsible for Huntington’s disease in important brain areas of non-human large primates. These positive preclinical results, together with other ongoing studies, are expected to support the submission of an investigational new…
Patients at advanced stages of Huntington’s disease have marked difficulties in controlling vehicles in a driving simulator compared to patients who are at a pre-symptomatic stage or healthy subjects, a study shows. The study’s authors think such driving simulators could be useful screening tools to aid clinicians in…
Signs of neurological dysfunction can start long before a clinical diagnosis of Huntington’s disease, a study shows. Early cognitive rehabilitation programs might be useful in the pre-manifest stage of the disease. The study, “Task-switching abilities in pre-manifest Huntington’s disease subjects” was published in Parkinsonism and Related Disorders. Although family history and DNA…
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