Angela Gritti from the San Raffaele Telethon Institute of Milan talks about the efforts of applying gene therapy to one of the most difficult body organs to access.
Category Archives: News
Aicardi-Goutières syndrome: when the immune system detects viruses where there are none
An interview with Anna Kajaste-Rudnitski from the San Raffaele Telethon Institute for Gene Therapy in Milan, author of a study that gives new insights into this rare syndrome and provides also useful information to investigate the interaction between the immune system and viruses, including Sars-CoV-2.
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.