Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…
News
Late-onset Huntington’s disease (emerging at age 59 or older), progresses similarly to common-onset Huntington’s except that late-onset patients more frequently showed gait and balance problems as their first symptom, new research has found. This finding suggests that observing problems with gait and balance might be helpful in diagnosing late-onset Huntington’s disease…
There may be no cure yet for Huntington’s disease, but patients — and their families — can still do plenty to extend their life expectancy through proper diet, exercise, and emotional support. That’s the message Norway’s Astri Arnesen wants to share with the world. Arnesen, president of the European…
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…
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…
#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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