Speranza contro esagerazione: alla ricerca della verità nei recenti titoli di Prilenia
Disclaimer: I have written this piece from a position of privilege – as an HD family member that has been fortunate to receive an education that allows me to deeply understand the nuances of Huntington’s disease. I know what it means not only at the biological level, but also at the family level. I am […]
Mini cervelli coltivati in vitro fanno luce sulla malattia di Huntington e su come potremmo curarla
Stem cells grown in 3D in a research lab can mimic some features of Huntington’s disease (HD). They also hold promise for transplantation studies to potentially add back cells that are lost in HD. But what would happen to those new cells? Would they get along with the cells still in the brain that have […]
Progressi costanti da uniQure: dati promettenti per la fine dell’anno
Con l'avvicinarsi delle feste, una gradita notizia è arrivata il 19 dicembre in un comunicato stampa di uniQure. Gli ultimi dati degli studi HD-GeneTRX sull’AMT-130, una terapia genica sperimentale che abbassa l’huntingtina, mostrano che il farmaco sembra essere ancora sicuro nel corso di pochi anni. Dato che il numero dei partecipanti è molto ridotto, […]
Allacciate le cinture: la terapia genica AMT-130 sembra rallentare i segni della malattia di Huntington in uno studio clinico di Fase I/II
New data from uniQure, who developed a one-and-done gene therapy for Huntington’s disease (HD) called AMT-130, indicates that the drug is relatively safe and might be able to slow down signs and symptoms of HD. AMT-130 is currently under investigation in Phase I/II clinical trials in Europe and the US which are mainly focused on […]
Conferenza della Hereditary Disease Foundation (HDF) 2024 – Giorno 4
We’re back for the last day of the Hereditary Disease Foundation conference! Serendipitous finding? Up first is HDBuzz co-founder and editor emeritus, Jeff Carroll. Jeff’s lab studies HD in mice and cells in a dish and investigates different potential treatments. The first story Jeff is telling us about is developing tools that lower HTT. He’s […]
Conferenza della Hereditary Disease Foundation (HDF) 2024 – Giorno 3
We’re back for Day 3 of the Hereditary Disease Foundation (HDF) conference! First up is a session on RNA dynamics – what’s that?! Read on to find out! Different HTT forms have different effects Up first is Gill Bates, who will tell us about her work in understanding how somatic expansion causes disease and investigating […]
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