«The LCP is already altering the natural history of disease and in this way negatively affects mortality statistics. (faith.org.uk)
In the Phase 1 trial, patients in the high dose group demonstrated improvement in motor function and they had a decreased need for supportive care compared to the natural history of the disease. (sciencedaily.com)
According to natural history of the disease, patients require nutritional and respiratory support by 12 months of age, and are not able to swallow or speak effectively. (sciencedaily.com)