LIRH Annual Conference 2024 - Neurology and Genetics: Huntington, ALS, Ataxia among clinics, research and associative networks

Bogdan Balas
Bogdan Balas is the Vice President and Global Head of Medical Safety and Pharmacovigilance and the Interim Head of Medical Affairs at uniQure. Bogdan earned his medical degree from Carol Davila Medical School in Romania and completed his residency in Endocrinology at the C.I. Parhon Institute. He then pursued a post-doctoral fellowship in Diabetes at the University of Texas Health Science Center. After moving to Basel, Switzerland, he worked at Roche as a Clinical Science Specialist and later as a Medical Safety Leader in Neurology. He then joined Novartis in Global Medical Affairs before returning to Roche as a Senior Medical Director in Safety Science for Neurology and Oncology. In 2020, he became VP and Head of Medical Safety at Galapagos, where he enhanced medical safety management and led the Basel site during the coronavirus pandemic. Bogdan joined uniQure in March of 2024.

Brian Beers
Brian Beers is an Associate Vice President with PTC Therapeutics and the Global Program Lead for PTC518 - PTC Therapeutics’ Huntingtin mRNA splicing modifier under investigation for the disease modifying treatment of Huntington Disease. A 24-year veteran of the biotech research community, Brian has been involved in the design, implementation, and management of clinical trials in a variety of therapeutic areas and several orphan indications including Epidermolysis Bullosa Simplex, Duchenne muscular dystrophy, limb-girdle muscular dystrophy and most recently, Huntington’s Disease. Brian graduated from the University of Illinois with a bachelor’s in microbiology and chemistry and is an avid traveler, hiker, and golfer. He currently resides with his wife and two children in a small town outside of Nashville, TN, USA.

Father Bernardo
Father Bernardo was born in Florence in 1968 where he completed his university studies, graduating with a thesis dedicated to the early humanism of that city. Since 1996 he has lived in the Abbey of San Miniato al Monte, of which he has been the father since 2009. From that monastery he contributes to the spiritual and cultural life of Florence. In 2019 he preached the spiritual exercises to Pope Francis and the Roman Curia, whose meditations were included in a volume published by San Paolo. Piemme recently published a book written in the form of a dialogue with a mother who lost a child on the reasons for hope and the ways of consolation.

Carlo Casali is an Associate Professor at the Department of Surgical and Medical Sciences and Biotechnologies, “Sapienza” University of Rome, Polo Pontino and a consultant Neurologist of the Rehabilitation Unit, ICOT – Rehabilitation Institute, Latina, Sapienza University of Rome, Polo Pontino, Italy. He received degree in Medicine with honors from Sapienza University of Rome in 1980, post-graduate Specialisation in Neurology with honors from Sapienza University of Rome in 1984 and PhD in Neuroscience from Catholic, University of Rome in 1989. He authored more than 250 publications in international and national journals. His main fields of research are: neuromuscular and neurodegenerative disorders, neurogenetics.

Marco Castori is in charge of the Medical Genetics Unit of the IRCCS-Casa Sollievo della Sofferenza Foundation, where he is also the contact person for the regional rare diseases network. Dr. Castori is particularly interested in hereditary connective tissue pathologies and Ehlers-Danlos syndromes, for which he is Institutional Representative at ERN ReCONNET and ERN-SKIN. Dr. Castori is coordinator of the Disease Group Ehlers-Danlos syndromes and Hypermobility Spectrum Disorders at ERN ReCONNET.

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.

Barbara D'Alessio
President and CEO of the LIRH Foundation, she obtained a law degree from the Sapienza University of Rome, subsequently a Master's degree in Marketing Management and a second level Master's degree in Patients' Advocacy Management. Barbara has over 20 years of experience in the non-profit sector in terms of management, planning, communications and international relations. She has been active in the Huntington Community since 2012.

Maurizio Inghilleri is a neurologist, with a PhD in the pathophysiology of movement disorders. He is associate professor of neurology at the University of Rome - Sapienza. His expertise and interests are in the field of neurophysiology. His clinical and research activity concerns the human motor system, focusing on motor neuron diseases and other rare neuromuscular diseases such as peripheral neuropathies, myasthenia gravis, pelvic floor dysfunction and visceral-somatic reflexes in humans. He is director of the ALS and Rare Neuromuscular Diseases Center of the Umberto I University Hospital. He coordinates a multidisciplinary team with experience in the treatment of patients suffering from ALS and rare neuromuscular diseases. 

Meghan Miller
Meghan received her PhD in Biomedical Sciences, Pharmacology from the University of California, San Diego, USA. With a drive to focus on neuroscience and rare diseases, she moved to Basel, Switzerland where she began her drug discovery career at Roche Pharma, first as postdoc in the Neuroscience, Pharma Research and Early Development (pRED) team, and then as a project leader in the Rare Diseases group, also under pRED. In 2019, Meghan joined Skyhawk Therapeutics, where she is currently Executive Director of Translational Science, and a core member of the Huntington’s Disease clinical development team.

Umberto Sabatini Graduated in Medicine and Surgery, Prof. Sabatini 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.
 

Annalisa Scopinaro is a rare disease advocate since the early 2000s. Since 2009 she has collaborated with Uniamo, the Italian Federation of Rare Diseases Associations, of which she is currently President. She has been ePags Representative in ERN Ithaca since 2018. Dr. Scopinaro participates in national and regional institutional tables (i.e. National Rare Diseases Committee, Working Group for the implementation of Extended Newborn Screening, Abruzzo and Marche Coordinations). She is part of the Scientific Committee of the Rare newsletter of the Ministry of Health-ISS and of many Italian and European working groups. She writes articles and collaborates in the drafting of technical documents on rare diseases. Dr Scopinaro collaborates, together with FAVO representatives, in the work of the Parliamentary Intergroup for Rare and Oncological Diseases created in the 19th legislature.

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).
 

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.