On Youtube, you can watch the GFB scientific webinar organized on 25 November 2022.
Prof. Isabelle Richard presented the gene therapy projects for LGMDs developed by Atamyo Therapeutics (LGMD2I/R9, LGMD2A/R1, LGMD2B/R2, LGMD2C/R5, LGMD2D/R3)
The webinar was held in English only but subtitles are available.
On Youtube, you can watch the GFB scientific webinar organized on 15 November 2022.
During this GFB science webinar, Prof. Volker Straub (University of Newcastle, UK) provides an overview of LGMD patient registries and updates on ongoing gene therapy projects for LGMD. The webinar was held in English only but subtitles are available.
GFB will participate in the AIM 2022 congress organized by the Italian Association of Myology. The congress will be held in Matera, Italy from the 19th to the 22nd October.
Lucica Bianchi (GFB Head of the communication office and social media manager) will present the poster "Observational study: the quality of life of patients with alpha-sarcoglycan, beta-sarcoglycan and gamma-sarcoglycan gene mutation" and will participate in the Round Table session: Meeting With Patients Associations scheduled for Saturday 22 October.
Journey is a clinical outcomes assessment study, also referred to as a natural history study. The study does not involve the use of an investigational study drug but rather studies the natural progression of the disease and how it affects the muscles, lungs, and heart in individuals receiving the current standard of care with their doctor/care team. The study is designed to collect medical history, clinical tests, quality of life questionnaires, and functional assessments on individuals affected with Sarcoglycan Limb-girdle muscular dystrophy.
Four centres are now open in Europe for the Natural history study JOURNEY:
GERMANY
Zentrum für Kinderheilkunde und Jugendmedizin Uniklinikum Giessen Marburg (UKGM), Standort Giessen
Giessen, Hessen, Germany, 35392
Contact +49 (0)641-985-56843 This email address is being protected from spambots. You need JavaScript enabled to view it.
Principal Investigator: Andreas Hahn, MD
ITALY
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy, 20122
Contact 39-02-55033802 This email address is being protected from spambots. You need JavaScript enabled to view it.
Principal Investigator: Giacomo Comi, MD
TURKEY
Lokman Hekim Etlik Hastanesi
Ankara, Turkey, 06100
Contact 90 532 234 12 26 This email address is being protected from spambots. You need JavaScript enabled to view it.
Principal Investigator: Haluk Topaloglu, Prof
UNITED KINGDOM
Institute of Genetic Medicine, International Centre for Life
Newcastle Upon Tyne, United Kingdom, NE1 3BZ
Contact 0191 241 8652 This email address is being protected from spambots. You need JavaScript enabled to view it.
Principal Investigator: Jordi Diaz-Manera, MD, PhD
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