Orchard Therapeutics Receives EC Approval for Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)
First gene therapy to receive full EU marketing authorization for eligible MLD patients. One-time treatment with Libmeldy has been shown to preserve motor and cognitive function. Achievement shared with research alliance partners Fondazione Telethon and Ospedale San Raffaele.
A novel finding on Kabuki syndrome, a rare genetic disease
The research, coordinated by the University of Trento, has recreated for the first time the pathological condition in a test tube discovering what happens in the cell nucleus and what hinders the formation of cartilages and bones.
Awarded Telethon Farmindustria Prize to three young researchers
Farmindustria and Fondazione Telethon have awarded three prizes of 10,000 euros to young researchers who work in the neonatology field.
Fondazione Roche rewards a young researcher of Tigem in Naples
Funded the research project by Paolo Grumati dedicated to the study of a neurodegenerative disease that causes generalized sensory loss.
Potential adverse reaction to gene therapy in a patient treated with Strimvelis for the treatment of ADA-SCID
Fondazione Telethon has recently learned that a patient suffering from a rare immunodeficiency of genetic origin, ADA-SCID, and treated in 2016 with gene therapy has developed leukemia.
Orchard Therapeutics Receives Positive CHMP Opinion for LibmeldyTM for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)
First therapy recommended for EU approval for eligible patients with confirmed diagnosis of late infantile or early juvenile MLD variants.
Osteopetrosis: new opportunity of treatment thanks to a licensing agreement
SiSaf announces a research collaboration and licensing agreement with the University of L’Aquila for the treatment of the debilitating bone disease, Autosomal Dominant Osteopetrosis Type 2.
Discovery of a novel drug candidate to develop effective treatments for brain disorders
Researchers at IIT-Istituto Italiano di Tecnologia discovered a novel chemical compound, which has the potential to became a new drug for the treatment of core symptoms of brain disorders like Down syndrome and autism.
Study of genetic condition leads to discovery of mechanism that promotes the formation of renal cysts and tumours, thanks to the support of Telethon and AIRC
At the Telethon Institute of Genetics and Medicine (Tigem) in Pozzuoli, the team led by Andrea Ballabio has described in Nature* how the study of a rare genetic condition has provided the key to understanding a mechanism that leads to the formation of cysts and tumours in certain organs, in particular the kidneys. Supported by Fondazione Telethon, Fondazione AIRC for cancer research and Regione Campania, the study included the participation the European Institute of Oncology (IEO) and the Institute of Cell Biology at the University of Innsbruck