Taube Philanthropies has donated $3 million to fund a research team using gene editing and stem cell techniques to develop treatments for Huntington’s disease. The team is composed of Dr. Matthew Porteus and Dr. Frank Longo of Stanford Medicine, and Dr. Steve Finkbeiner of the Gladstone Institutes, said a Stanford press…
Disruption in the trafficking of molecules in and out of the cell’s nucleus may contribute to the pathology of Huntington’s disease, according to results of recent research. The study “Mutant Huntingtin Disrupts The Nuclear Pore Complex” was published in the journal Neuron. Huntington’s disease is caused by mutations in…
Stealth BioTherapeutics has started a Phase 1/2 clinical trial to investigate the safety and effectiveness of SBT-20 in patients with early-stage Huntington’s disease in the Netherlands. SBT-20 was designed to restore the proper activity of mitochondria, the cell’s powerhouses, in Huntington’s patients. Mitochondria organelles become faulty as the disease progresses.
Treatment with Vaccinex’s experimental antibody VX15 was seen to stop loss of brain volume and activity in patients with late prodromal (pre-manifest) and early manifest Huntington’s disease, according to preliminary results of the SIGNAL clinical trial. VX15 is an antibody that inhibits a protein called semaphorin 4D (SEMA4D), which participates…
The latest results on the effectiveness and safety of pridopidine (called Huntexil in Europe) for the treatment of Huntington’s disease (HD) will be presented at the annual meeting of the American Academy of Neurology (AAN) April 22-28 Boston. Pridopidine is an investigational drug that has the potential to…
Pope Francis will meet May 18 in the Vatican with patients and others who are fighting Huntington’s disease in what organizers are billing as the world’s largest gathering ever of the Huntington’s community. The event is part of Huntington’s advocates’ HDennomore global initiative. The phrase, which means Hidden No…
Probiodrug AG recently announced the results of a preclinical study investigating the role of glutaminyl cyclases (QCs) in Huntington’s disease. The results will be presented at the 12th Annual HD Therapeutics Conference of the CHDI Foundation, in Malta, April 24-27. Probiodrug AG is a biopharmaceutical company developing innovative…
A recent report showed that patients with early Huntington’s disease (HD) cannot control their impulsiveness. However, when confronted with a risk-taking situation, they present a similar response as individuals without the disease. Recognizing this behavior pattern can have important implications during treatment and rehabilitation. The study, “Early Huntington’s Disease:…
A virus that delivers UniQure‘s gene therapy candidate AMT-130 has done a good job of getting the Huntington’s disease treatment into the brains of primates, company research shows. Injections of the virus delivered the treatment with precision and without side effects, researchers said. AMT-130’s delivery component is an AAV5 viral vector. AAV5…
Teva Pharmaceutical Industries‘ Austedo tablets have received U.S. approval for treating chorea, or severe movement problems, associated with Huntington’s disease (HD). Austedo (deutetrabenazine, SD-809) is the first deuterated drug the U.S. Food and Drug Administration (FDA) has authorized, and only the second therapy it has approved for Huntington’s. The FDA had…
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