תפריט
Welcome to the anniversary online conference of little steps association for children with Duchenne/Becker Muscular Dystrophy
The conference this year (2020) included 5 sessions and around 40 lectures from world wide experts regarding clinical trials update, standard of care and tools to deal with everyday life.
For more information see the full programme attached.
Research
- Loaded Exosomes as a Novel Approach for Gene Therapy in Duchenne Muscular Dystrophy
- The Importance of Physical Activity in Duchenne and Becker Muscular Dystrophy – a Physiological Research
- Introduction to DMD Therapies
- CRISPR/Cas 9 for DMD
- An IGNITE DMD Update, Solid Bio
- ASP0367 Development Program Update
- DMD Genethon International Program: Natural History and Future Gene Therapy Trial
- FibroGen DMD Program. Pamrevlumab LELANTOS Phase 3 Studies in Patients with DMD
- Italfarmaco Givinostat Development Program Update
- Micro-dystrophin Gene Therapy
- Pfizer Gene Therapy Trial (PF-06939926) for Duchenne Muscular Dystrophy
- Outcomes and Learnings from The Sideros Interim Analysis
- Safer steroidal anti-inflammatory? Structural to Clinical Studies of Vamorolone
- Update on ATL1102 Program for Non-Ambulant Boys with DMD
Treatment
Living with Duchenne
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