4 Dicembre 2017 - Con il Premio, quest'anno alla sua I Edizione, la Fondazione intende valorizzare i ricercatori italiani che svolgono le migliori ricerche, cliniche o biologiche applicate, pubblicate o in corso di pubblicazione, su riviste scientifiche indicizzate.
June 6, 2015 - The next conference on Neurological Clinical Trials will be held in Milan the next June, 25. A focus on Huntington Disease will be presented by Prof Ferdinando Squitieri.
In a recent community letter sent out on January 26, Prilenia Therapeutics announced that people who took part in the PROOF-HD phase three trial will be able to take pridopidine, the experimental drug administered as part of this study, thanks to a expanded access/compassionate use program (EAP/CUP).
An important study has led to the development of a cognitive assessment tool that could help predict the severity of Huntington disease (HD) in those close to onset.
Huntington disease (HD) - a condition that affects one’s movement, cognition, and behavior—manifests fully in adulthood.
GLUT-1, a protein that transports sugars into human tissue cells, is deficient in the brain and peripheral tissues of children with Huntington's disease, but not in adults.
Breakthrough in the study of Huntington's disease: an alteration in the pediatric HD is not present in the adult HD
Our brain consumes over a quarter of the energy needed to make our entire body function, translated into approximately 200-300 kilocalories per day. Without sugars, it would not be able to function properly and support very important functions such as the activity of neurons and the processing of information.
PTC518 is a small molecule capable of reducing the protein that causes Huntington's disease.
Pivot-HD: new hope for the treatment of Huntington Disease
PTC518 is a small molecule capable of reducing the protein that causes Huntington's disease (HD).
Pridopidine did not show benefits on functional capacity in people with Huntington's disease
Pridopidine has a long history. The drug was initially tested by the NeuroSearch company which believed that it had an effect on dopamine as its main mechanism and was therefore able to act on movement coordination. On this assumption, starting from 2007, two studies were conducted, MermaiHD and HART, both failed.
In 2012, Teva Pharmaceuticals bought the right to test pridopidine again and promoted a third study, PRIDE-HD, which tested different doses, again with the aim of verifying the drug's effects on motor function.
An update on therapeutic trials for the treatment of Huntington's disease.
The LIRH Foundation Annual Conference back in attendance.
The last five years have led to great milestones in the history of Huntington's disease research. At least three can be highlighted:
1) The setting up of the largest research network ever created for a rare disease, capable of connecting researchers from all over the world;
2) The launch of experimental therapies potentially capable of modifying the course of the disease by directly affecting its cause;
As always, we are closely following the developments of the HD ongoing trials. Unfortunately, not too encouraging news arrives on the studies of Novartis (with branaplam) and UniQure (gene therapy with AMT-130).
As always, we are closely following the developments of the HD ongoing trials. Unfortunately, not too encouraging news arrives on the studies of Novartis (with branaplam) and UniQure (gene therapy with AMT-130). Both pursue the strategy of huntingtin lowering, both mutated and normal length - with different tools - to counter Huntington's disease.
uniQure announced the dosing of the first two patients in its European open-label Phase Ib/II clinical trial of AMT-130, a potential one-time gene-therapy approach for the treatment of Huntington’s disease. The clinical trial is taking place at several sites in Poland, the United Kingdom and Germany.
On the last February 2, 2022 uniQure sent out a press release with new updates on their gene therapy for Huntignton Disease.
