PTC Therapeutics recently published an article in the prestigious journal Nature Communications describing the functioning of a series of small molecules, that can be taken orally, and are able to distribute themselves in the brain and also in the rest of the human body.
Reducing levels of huntingtin, the protein that, when mutated, causes Huntington's, was the first strategy designed to directly target the cause of the disease.
However, the current approaches have some limitations, as the molecules under study:
Thanks to the children met in the context of the 'Space Huntington' project, it was possible to grasp and describe the signs of HD much earlier than we have seen so far.
The recent fase III Generation HD 1 study data discloure from Roche was accompanied by the announcemenet of re-testing the drug in a Fase II study on a subgroup of patients.
A few days ago Roche announced that a Phase II study with tominersen will start soon. Tominersen is the ASO (Oligonucleotide Anti Sense) whose experimental administration was interrupted in March 2021, following the negative evaluation of its effects on patients, by an Independent Evaluation Commission. In this article we take stock of what we know and what we don't know yet.
Time spent on hobbies throughout life helps to better cope with loss of cognitive function and independence in people with early stage Huntington's disease.
The study, entitled Cognitive Reserve in Early Manifest Huntington Disease Patients: Leisure Time Is Associated with Lower Cognitive and Functional Impairment, was recently published in the Journal of Personalized Medicine, in the special issue on Huntington's disease, edited by prof. Ferdinando Squitieri.
It was conducted by the IRCCS Casa Sollievo della Sofferenza and by the LIRH Foundation, in collaboration with the Sant’Andrea Hospital in Rome and the IRCCS Carlo Besta in Milan.
Poco meno di un anno fa si assisteva all'insuccesso di tre studi clinici sulla malattia di Huntington. Oggi, per fortuna, possiamo parlare di nuove prospettive terapeutiche, basate su una tecnologia più evoluta delle precedenti.
Poco meno di un anno fa siamo rimasti tutti sgomenti di fronte all'insuccesso di tre studi clinici, ma oggi non solo possiamo parlare di nuove prospettive terapeutiche, ma addirittura di prospettive basate su una tecnologia più evoluta delle precedenti.
Per arrivare a questo, c’è stato un enorme impegno da parte di tutti: le industrie, i ricercatori e i pazienti. Non dobbiamo mai dimenticare che è possibile condurre sperimentazioni sulla malattia grazie all'elevato tasso di partecipazione dei pazienti.
16 December, 2021: The data support the tolerability of AMT-130, a potential one-time gene-therapy approach for Huntington’s disease
Yesterday, December 16, 2021, uniQure sent out a press release whose 4 highlights are as follows:
1 - The treatment was well tolerated with no significant safety issues related to AMT-130 in first two treated patients through one year of follow-up;
2 -Neurofilament Light Chain (NfL) rose, as expected, immediately following surgery and returned to baseline in treated patients;
Pharmaceutical companies confront researchers and patients on the HD treatments perspectives
The future of therapeutic research to cure Huntington's disease.
This is the title of the LIRH Foundation 2021 Annual Conference, which will take place online next Friday 3 December, from 15.00 to 17.30 CET.
Thanks to artificial intelligence, a drug has been discovered to be tested in the treatment of the "chorea" symptom in Huntington's disease: it is called SOM3355
Thanks to artificial intelligence, a drug has been discovered to be tested in the treatment of the "chorea" symptom in Huntington's disease: it is called SOM3355
The US Food and Drug Administration has just granted orphan drug designation to SOM3355, an investigational drug for the treatment of chorea (involuntary jerking movements) in patients with Huntington's disease.
A recent study confirmed epidemiology studies on HD conducted in different Countries, including the one conducted by LIRH in Italy in 2016.
A recent study confirmed HD epidemiology data in different Countries, including Italy, were the research was carried out by LIRH Foundation in 2016.
The results of a study describing a link between specific changes in behavior and cognitive abilities in patients with Huntington's disease have been published in the journal Brain and Behavior.
Open The results of a study describing a link between specific changes in behavior and cognitive abilities in patients with Huntington's disease have been published in the journal Brain and Behavior. configuration options
The results of a study describing a link between specific changes in behavior and cognitive abilities in patients with Huntington's disease have been published in the journal Brain and Behavior.
