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.
Finally Good news for the Huntington's community!
We received yesterday the official communication from uniQure, announcing that the enrollment of the first patient cohort in the study of ATM-130 for the treatment of Huntington's disease has concluded, ahead of schedule.
But the good news don't end here: a new clinical trial with AMT-130 will be initiated in Europe by the end of the year.
26/03/2021
On Wednesday, 24/03/2021 was held a meeting via zoom between investigators, associations and patients to answer the many questions arising from the unexpected news of the interruption of the dosing of the experimental drug tominersen in the phase III 'Generation HD1" clinical trial.
The investigators of the Italian centers involved in the study, promptly responded to the need for immediate answers, taking into account that the data behind this decision have not yet been made public.
23/03/2021
On March 22nd, 2021, a press release from Roche revealed that the phase III study of tominersen had been halted on the advice of the Independent Data Monitoring Committee (iDMC).
