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Orchard Therapeutics PLC -- ADR (NASDAQ: ORTX) -- Phase 2 data for OTL-200 in metachromatic leukodystrophy
Orchard Therapeutics announced results from an integrated data analysis of OTL-200, a gene therapy in development for the treatment of metachromatic leukodystrophy at the San Raffaele-Telethon Institute for Gene Therapy.
The oral presentations will highlight data from the company's neurometabolic portfolio, including investigational candidates OTL-203 for mucopolysaccharidosis type I (MPS-I) and OTL-200 for metachromatic leukodystrophy (MLD).
Alfie, of Longlands, died peacefully in his mam Debbie's arms on June 13, having battled the rare neurological condition Alexander's Leukodystrophy since he was a toddler.
Dougie was devastated when his six-month-old grandson Tyler McQueen, of Dumfries, passed away on January 7 - just weeks after the tot was diagnosed with rare brain disease Leukodystrophy.
As a result, two known mutations (c.114C>T; p.Arg372Trp and c.418dupC; p.His140Profs36*) were identified in the ARSA gene that led to metachromatic leukodystrophy (MLD) with combined heterozygosity.
Les Martin's world was turned upside down when Cathal, five, and three-yearold Ciaran were diagnosed with metachromatic leukodystrophy - an illness for which there is no cure.
The company plans to enroll 12 patients in the ongoing Phase 2 study in inherited metabolic disorders, which include cALD, Hurler syndrome, metachromatic leukodystrophy and globoid cell leukodystrophy.
The firm plans to enrol 12 patients in the ongoing Phase two study of inherited metabolic disorders including cALD, Hurler syndrome, metachromatic leukodystrophy and globoid cell leukodystrophy.
###CLN6###Neuronal Ceroid Lipofuscinosis-6###PYCR2###Hypomyelinating Leukodystrophy, 10