Restoring the peroxisomal fission protein PEX11β improved motor and cognitive deficits and mitochondrial gene expression in a toxin-induced mouse model of Huntington's-like disease, with Yin Yang 1 identified as a key regulator of PEX11β and Complex II co-expression. In a study published in…
IXICO PLC (LSE:IXI, OTC:PHYOF, FRA:PYPB), the brain imaging analytics company, has presented research suggesting that reducing differences between MRI scanners does not always make it easier to track Huntington's disease. The work was shown at the European…
Key Highlights from the Publication Introduction of a computational model capable of predicting the impact of SI-targeted therapies in HD across a spectrum of inherited CAG-repeat lengths and potential ages of therapeutic intervention. Quantitative correlations that link levels of MSH3 knockdown…
Newswise — NEW YORK, Oct. 28, 2026 -- The Huntington's Disease Foundation (HDF) has awarded $2 million for its 2026 Transformative Research Awards (TRAs) to support two exceptional research collaborations with the potential to advance treatments for Huntington's disease (HD). Made possible by the support of…
Using genetic engineering technology, a team of scientists has established a pig model of Huntington's disease (HD), an inherited neurodegenerative disease. The researchers anticipate that the pigs could be a practical way to test treatments for HD, which is caused…
New data showing rapid and durable full-length MECP2 expression in vivo with single-stranded AAV support improvements observed in NGN-401 Phase 1/2 trial New in vivo data demonstrate that miRNA-based auto-regulatory technology is not responsive to endogenous MeCP2 levels on a cell-by-cell basis In…
The findings support the clinical benefits of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD). Patients with Duchenne muscular dystrophy (DMD) aged 8 and 9 years experienced significant functional benefits with delandistrogene moxeparvovec-rokl (Elevidys; Sarepta Therapeutics) in part 2 of the EMBARK study (NCT05096221), according…
Alnylam Pharmaceuticals, Inc. (ALNY) RNAi Roundtable: Exon 1 Targeting With Brezosiran for Huntington's Disease October 26, 2026 10:00 AM EDTCompany... Alnylam Pharmaceuticals, Inc. (ALNY) RNAi Roundtable: Exon 1 Targeting With Brezosiran for Huntington's Disease October 26, 2026 10:00 AM EDT Joshua Brodsky - Vice President of Investor Relations Hello,…
A population-based surveillance study found that China’s age-standardized congenital anomaly mortality rate fell from 2008 to 2021, while relative regional inequalities increased for several causes among children under five. Congenital anomaly mortality in China declined between 2008 and 2021, but a population-based surveillance study…
May 20, 2014, 08:30 ET With support from the Griffin Foundation in Washington, D.C., and in partnership with MedStar Georgetown and The Huntington's Disease Society of America, the British-based Huntington Disease Youth Organization (HDYO) has opened its first U.S. office at MedStar Georgetown. "HDYO will…
Gene therapies that add or fix genes with AAV, lentiviral and other vectors: hearing loss, SMA, Duchenne, hemophilia and eye disease, with their trials, approvals and safety.
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