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

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.

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.

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).

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.

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.

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.