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…
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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…
The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow Macedonians with rare diseases were so daunting, they would have scared off all but the most determined. At that time, few doctors in the developing country of 2 million — now called North Macedonia — had…
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing problems as Alport syndrome. Although she had known for years that something was wrong, the news that David would battle a lifelong rare disease devastated…
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Encouraging Brain Donations for Huntington’s Research Goal of HDSA and CHDI Foundation Partnership
Encouraging the families of people affected by Huntington’s disease to donate, post-mortem, their brain and other organs so as to advance research into the disease and its possible treatments is the goal of the Huntington’s Disease Society of America (HDSA) and the CHDI Foundation partnership. Called HD Legacy, the…
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…
Measuring the seeding activity of mutant huntingtin (mHTT) protein — its ability to trigger protein aggregation — could be an early biomarker of Huntington’s disease with the potential for monitoring disease onset and progression, according to preclinical findings in mouse and fly models. The study, “mHTT Seeding…
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…
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