News from Research
01 / 01
First gene therapy to receive full EU marketing authorization for eligible MLD patients.
Lorenzo wants to go far
“I can’t walk”. When you hear those words on the lips of a child below the age of three, it’s hard to turn your back and walk away. Imagine you’re the child’s parents. The words came out with the composed calmness of a fact of nature.
Against mitochondrial diseases together with Andrea
Knowing that the machine you need to breathe with has just one hour’s run time doesn’t make life very easy!
In Italia, una nuova speranza
Dopo una lunga attesa, nel 2001, Virginia e Rafael festeggiano la nascita del loro piccolo Rafael Enrique. Poco dopo la nascita, però, inizia il dramma di questa famiglia venezuelana: il bambino si ammala spesso per gravi infezioni.
An important contribution to the elucidation of the mechanisms through which blood stem cells arise during embryonic life from a study conducted by the San Raffaele Telethon Institute for Gene Therapy.
Gene therapy targeted to the brain: a complex but not impossible challenge
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.
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.