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.
Tag Archives: luigi naldini
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.
From Telethon-funded gene therapy comes a new weapon in the battle against cancer
The innovative technique developed in the laboratories of the Telethon San Raffaele Institute for Gene Therapy for the treatment of two extremely serious genetic diseases can also be effective in treating tumours.