Huntington’s
Smartphone assessments could help researchers more precisely detect progression in Huntington’s disease, potentially improving the efficiency of clinical trials, new research suggests. A five-minute test delivered via a custom app calculates a Huntington’s Disease Digital Motor Score (HDDMS) using five simple assessments of movement control – including balance, finger tapping and involuntary movements. Huntington’s disease [...]
Gene therapy company uniQure recently announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington’s disease. There are currently no disease-modifying therapies available for Huntington's Disease, highlighting the urgent need for innovation. uniQure has stated that AMT-130's Breakthrough Therapy designation has been granted based on clinical [...]
Results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disease (HD) patients demonstrated that the treatment met its primary endpoint of reduction in blood Huntingtin (HTT) protein levels. The treatment showed a reduction in HTT protein levels at Week 12 and favourable safety and tolerability. The 12-month [...]
Researchers have uncovered two proteins that impact Huntington's Disease - one which mitigates the condition and another which exacerbates it. A decade ago, University at Buffalo researchers shed some light on an enduring neuroscience mystery: How exactly does a mutated huntingtin protein (HTT) cause Huntington’s disease? They found that HTT is something like a traffic [...]
For the first time in 35 years, the number of people in northern Scotland who have the Huntington's gene has been counted, revealing that the region has one of the highest rates of the disease in the world. The research, from the University of Aberdeen in partnership with NHS Grampian, found that confirmed that rates [...]
New research shows that the adult brain can generate new neurons that integrate into key motor circuits - demonstrating that stimulating natural brain processes may help repair damaged neural networks in Huntington’s and other diseases. It was long believed that the adult brain could not generate new neurons. However, it is now understood that niches [...]
The University of Leicester and Leicestershire Partnership NHS Trust Huntington's Disease Network is celebrating its one-year anniversary this April. The Leicester Huntington’s Disease Network (LHDN) was established in April 2024 to help drive community support, interventions and engagement for those living with Huntington’s disease. The network is co-led by lecturer and practising clinical psychologist Dr [...]
Biotech company Skyhawk Therapeutics has developed a novel SKY-0515 small molecule RNA splicing modulator targeting Huntington's Disease. SKY-0515 is an investigational small molecule RNA splicing modifier developed through the Skyhawk's novel RNA-splicing platform. SKY-0515 is designed to reduce both HTT protein and PMS1 protein, an additional key driver of somatic CAG repeat expansion and HD [...]
A biotechnology firm working to correct an underlying cause of Huntington's disease will announce positive data this week on its ongoing gene therapy development. Latus Bio aims to address mutations in the HTT gene which lead to expansion of >39 CAG trinucleotide repeats which cause Huntington's. A CAG repeat is a sequence of DNA in [...]
A life sciences firm aiming to harness the RNA molecule that carries genetic instructions to treat Huntington’s has been boosted by a major fundraise. Atalanta Therapeutics, from Boston, US, has raised Us$75m from investors to help fund its groundbreaking search for genetics-based treatments for neurological diseases. RNA is a molecule that carries genetic instructions from [...]
