A novel class of proteins, dubbed heat-resistant obscure (Hero) proteins, can protect other proteins from a variety of cellular stresses, and also can prevent the formation of protein aggregates typical of Huntington’s disease and amyotrophic lateral sclerosis (ALS). The new protein class was described in the study “…
Vaccinex’s Phase 2 SIGNAL study, evaluating the investigational antibody pepinemab (VX15/2503) in Huntington’s disease patients with late prodromal and early disease, is ongoing and has tested pepinemab in all 265 patients enrolled in cohort B, the company announced. As of March 31, only 37 patients in this group…
While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…
Teva Pharmaceuticals’ Austedo (deutetrabenazine tablets) has been approved by China’s National Medical Products Administration (NMPA) for treating chorea associated with Huntington’s disease and tardive dyskinesia…
European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…
Multiple government agencies have cooperated to allow MMJ International Holdings to import cannabis extracts to the United States, for use in potential therapies the company is developing for clinical trials in Huntington’s disease and multiple sclerosis. The cannabis plant makes numerous compounds called cannabinoids, two of the…
Because of the COVID-19 pandemic, Huntington’s Disease Awareness Month organizers have come up with creative ways to show support and call attention to the disorder during May. In past years, May has been full of fundraising and educational activities such as outdoor bake sales, group walks and runs, golf…
The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…
AskBio announced that it has acquired BrainVectis, a company developing gene therapies for Huntington’s disease and other neurodegenerative conditions. The two companies will work to advance BrainVectis’ gene therapy programs for neurodegenerative diseases associated with problematic fat metabolism in the brain, including a lead candidate therapy for Huntington’s called…
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