LIRH Annual Conference 2023 - New hopes from research for Huntington's disease

The LIRH Foundation's appointment to update you on the developments of clinical research on Huntington's disease is in Rome on Saturday 2 December 2023. Participation is free, but registration is required for organizational and logistical reasons.



Convegno Annuale LIRH 2023 - Nuove speranze dalla ricerca per la malattia di Huntington
Data e ora
6 hours 30 minutes
Add to calendar 2023-12-02 09:30:00 2023-12-02 16:00:00 Convegno Annuale LIRH 2023 - Nuove speranze dalla ricerca clinica sulla malattia di Huntington Convegno Annuale LIRH 2023 - 41.9122289, 12.5153977 LIRH - Lega Italiana Ricerca Huntington e malattie correlate onlus Europe/Rome public
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Type of event
Convegno annuale
Free access
Max number of partecipants
Tickets available
Ancora pochi disponibili
Centro Congressi Roma Eventi, Piazza della Pilotta 4, Roma

41.9122289, 12.5153977


program & speakers


Clinical trials update session

convegno lirh 2021 barbara d'alessio

Barbara D'Alessio

President and CEO of the LIRH Foundation, graduated in law from the Sapienza University of Rome, Master in Marketing and Master in Patients Advocacy Management from the Cattolica University in Rome. She has over 20 years of experience in the non-profit sector. She has been active in the Huntington Community since 2012.


convegno lirh 2023 Umberto Sabatini

Umberto Sabatini

Graduated in Medicine and Surgery, he has a specialization in Neurology and Radiology. He acquired a European PhD in Neuroscience from the University of Toulouse, France, an Italian PhD in Neurobiology from the University of Catania. He is a full professor at the Department of Medicine and Surgery of the Magna Graecia University and director of the U.O.C. of Neuroradiology at the Mater Domini university hospital in Catanzaro. He has clinical experience in the field of Neuroradiology as well as research experience in the field of structural and functional images of the brain with magnetic resonance imaging in diseases of the nervous system. He has been teaching at qualified European universities. He was and is responsible for national and international research projects. He has participated in clinical trials (phase II and III) in cerebral ischemia, multiple sclerosis, Alzheimer's disease, Parkinson's disease, Huntington's disease and multiple system atrophy.


convegno lirh 2022 ferdinando squitieri

Ferdinando Squitieri

Ferdinando Squitieri is a neurologist with a PhD in Neurobiology. He has been studying and treating patients with HD and juvenile onset HD (JoHD) over the last 30 years. He is Head of the Huntington and Rare Diseases Unitat Fondazione IRCCS Casa Sollievo Sofferenza Research Hospital in San Giovanni Rotondo and Head of the Neurology Departmentat CSS-Mendel Institute In Rome. He Is also Co-founder and scientific officer of Lega Italiana Ricerca Huntington (LIRH) Foundation and associated researcher of the Italian National Research Council (CNR).



New Hopes

convegno lirh 2022 Toon van Gool

Toon van Gool

Toon van Gool is an Executive Director with PTC and Head of Commercial Development and leading the commercial strategic development of PTC518 - PTC Therapeutics’ Huntingtin mRNA splicing modifier under investigation for the disease modifying treatment of Huntington Disease. With 28-years of experience in the pharma and biotech industry, Toon has been involved in the development and launch strategies of a variety of therapeutic areas and several orphan indications including chronic iron overload in patients with Beta Thalassemia, Chronic Lymphocytic Leukemia, Cystic Fibrosis, Duchenne Muscular Dystrophy, and most recently, Huntington’s Disease. Toon has his Masters in Biopharmaceutical sciences from the University of Leiden, the Netherlands and his MBA from Bradford University UK and is an avid traveler, hiker, and skier. He currently resides with his wife and three children in a small town central In Switzerland.


convegno lirh 2023 David H. Margolin

David H. Margolin

David H. Margolin, MD, PhD, is Vice President for Clinical Development at uniQure Inc., and leads uniQure’s HD gene therapy program. A neurologist formerly on staff at Harvard-affiliated hospitals in Boston, Dr. Margolin has designed and led clinical trials across a range of neurologic, psychiatric, and rare disease indications. His laboratory and clinical research studies have been published in prestigious medical journals and led to drug approvals in over 60 countries.


convegno lirh 2023 Andres Cruz-Herranz

Andrés Cruz-Herranz

Andrés Cruz-Herranz is a neurologist and neuroscientist. He has been involved in research and development of neuroprotective therapies in neurodegenerative diseases for eleven years. Andrés is currently medical director of clinical development at Prilenia Therapeutics.



Renewed hopes

convegno lirh 2021 Asela Bandara

Asela Bandara

Asela studied medicine at Queen Mary University of London specialising in Internal Medicine (MRCP) followed by certification in Pharmaceutical Medicine (MFPM). Within industry, he worked almost exclusively on rare disease clinical development with Sanofi Genzyme, Healx a UK-based AI biotech startup and Wave Life Sciences, having joined the team as a Sr. Medical Director on the HD and DMD programs.


convegno lirh 2023 Lauren Boak

Lauren Boak

Lauren Boak received training in biomedical science and research in Australia. She moved to Switzerland over 16 years ago to work for the pharmaceutical company Roche, where she has worked on many different clinical programs in neuroscience. Lauren has been working on the tominersen program for the past 6 years, being constantly inspired by the strength and resilience of the HD community.



Q&A, Final Discussion and Conclusions




HD Associations Session

convegno lirh 2022 Alessia Migliore

Alessia Migliore

Alessia is from an HD family. She is co-founder and currently President of NOI Huntington, The Italian Youth HD Association. She works as a Psychiatric Rehabilitation Therapist at the Mental Health Department in Naples.


convegno lirh 2022 nicolo zarotti

Nicolò Zarotti

Nicolò Zarotti is an Academic and Clinical Psychologist. He completed his undergraduate (BSc) and postgraduate (MSc) degrees in Psychology at the University of Trieste (Italy). He moved to the UK in 2013, where he went on to complete a PhD at Lancaster University and a Doctorate in Clinical Psychology (ClinPsyD) at the University of East Anglia. Over the past 10 years, he has been carrying out research aimed at developing psychological and neuropsychological approaches to mental health and cognitive difficulties in people with motor neurodegenerative conditions such as Huntington’s disease, Parkinson’s, motor neurone disease, and multiple sclerosis.


convegno lirh 2022 Giusepe Scandale

Giuseppe Scandale

Part of the ‘LIRH family’ since many years. President of LIRH Toscana, HD family association. Aware of the power of information, that helps many families learn about HD and the available treatments options, we promote a lot of dissemination initiatives. Proud to have approached many HD hidden families, who then reached out our Foundation.


convegno lirh 2022 Nicola MICCHETTI – Presidente LIRH Puglia

Nicola Micchetti

Nicola Micchetti, 76 years old, President of the LIRH Puglia Association, engineer, former Coordinator of the Technical Sector of the Autonomous Housing Institute of the Province of Bari and currently retired. “Having had my wife suffering from Huntington's disease, I had the opportunity to meet Prof. Squitieri and his team. I appreciated their strong expertise and great humanity, so I joined the LIRH family with the aim of spreading the knowledge of HD, to support the Association growth and to be of help for those who are in difficulty in finding solutions to welfare problems by public institutions in ordinary daily life”.


convegno lirh 2022 alessia zurru

Alessia Zurru

Degree in Biological Sciences at the University of Cagliari, Master’s in journalism and Communication of Science at the University of Ferrara. She lost her mom in 2015 because of HD. In 2018 she decided to take an active role in creating a Huntington network that would bring together Sardinian families. In March 2022 Alessia co-founded the Association LIRH Sardinia.


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Locandina Convegno Annuale LIRH 2023978.49 KB 978.49 KB
Programma Convegno LIRH 20231.71 MB 1.71 MB