Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare…
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Stem cell therapy delivered via the nose led to longer lives and evidence of better circadian (internal “clock”) activity in a mouse model of Huntington’s disease, and may offer a way of using these treatments in people, a study reports. The study, “Intranasal Administration of Mesenchymal Stem Cells Ameliorates…
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage girls, and has the nation’s third-highest rate of uninsured residents — with 13.9% of all Oklahomans lacking health coverage. As if…
Treatment with EIP Pharma’s investigational therapy neflamapimod (VX-745) can restore nerve cells’ communications and prevent their progressive degeneration and death, new preclinical results show. All together these data further support neflamapimod’s use to treat several neurodegenerative disorders characterized by impairment in nerve…
Differences in the activity of a protein called Cdk5 in the brain could be a cause of — and perhaps even a target for the treatment of — depression related to Huntington’s disease, research in mice suggests. The study, “Cyclin-Dependent Kinase 5 Dysfunction Contributes to Depressive-like Behaviors…
EIP Pharma is launching a proof-of-concept Phase 2 clinical trial to assess the ability of therapeutic candidate neflamapimod to treat cognitive dysfunction in patients with early-stage Huntington’s disease. The trial (NCT03980938), which is currently recruiting, is taking place at the John Van Geest Centre for…
Emerald Health Pharmaceuticals announced that two of its cannabinoid-derived candidates — CBGA-Q and CBGA-Q-Na Salt — showed anti-inflammatory and neuroprotective effects in a mouse model of Huntington’s disease. However, these benefits were not superior to those obtained from treatment with the company’s already patented EHP-102 compound. The…
A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of treatments for the 95% of such illnesses that currently don’t have one. The European Joint Programme on Rare Diseases (EJP…
Egypt, the most populous country in the Arab world, has the highest prevalence of Huntington’s disease, yet this nation of 100 million is overlooked when it comes to research on the fatal genetic disorder. Huntington’s disease occurs in 2.7 per 100,000 inhabitants worldwide, and 10 per 100,000 in Europe,…
People with Huntington’s disease may have two different symptom profiles, a cognitive-motor and a psychiatric profile, associated with different clinical and neurobiological features. The findings of the study, “Specific patterns of brain alterations underlie distinct clinical profiles in Huntington’s disease,” were published in NeuroImage: Clinical. Huntington’s…