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Clinical trials on Huntington Disease. LIRH Annuale Conference: Rome, December 3, 2022

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;

3) The advent of new technologies for the identification of therapeutic targets and the creation of new assessment tools based on artificial intelligence and machine learning.

These goals represent a fundamental premise for a new perspective of care and an epochal change of strategy for medical research in general.

All these will be discussed in Rome on Saturday 3 December 2022 in the context of LIRH Foundation Annual Conference starting at 09.30 at Centro Congressi Roma Eventi in Piazza della Pilotta 4 (Trevi Fountain).

The national event is meant to update the HD community on the progress of clinical research, with particular attention to what is happening in Italy.

"2023 is likely to be as the year of the beginning of a new scientific era because, for the first time: drugs coming from a screening based on innovative Artificial Intelligence systems; drugs capable of selectively interfering with the genetic cause, sparing the part inherited from the healthy parent; drugs capable of having a direct effect against the genetic cause both in the brain and in peripheral tissues; drugs capable of significantly modifying the course of the disease are being tested simultaneously." these are the words of Ferdinando Squitieri, co-founder and Scientific Officer of the LIRH Foundation, Principal Investigator of all the main clinical studies on Huntington's disease.

A panel of dialogue with authoritative representatives from the regulatory authority (Sandra Petraglia, manager of the pre-authorization area of ​​AIFA), of the rare diseases  the world (Eva Pesaro, Vice President of Uniamo), of pharmaceutical companies, of the HD community, of researchers will confront on these topics. Selene Capodarca from the EHDN will also participate as a speaker.

"The possible treatment options that we see today for this very difficult rare disease are possible thanks to the collaboration between researchers and patients, to the greater awareness of patients about their role in the success of research programmes, not only as study protocols partiocipants, but also as actors of their drafting and development" – ​​declared Barbara D'Alessio, President and Executive Director of the LIRH Foundation.

A key role will be played by patients and their representatives, thanks to the presence of the Associations that are part of the LIRH network: LIRH Toscana, LIRH Puglia, LIRH Sardegna and NOI Huntington/La Rete Italiana dei Giovani, who will confront on patients needs in the afternoon.


For the detailed program, click HERE

The Conference will take place in presence, but it will be also possible to participate remotely by registering to the following LINK.