'Unmovable monster': The drug trial that changed how biotechs approach rare disease treatments

Billy Ellsworth was 10 years old when he started flying from his home outside Philadelphia to Columbus, Ohio to take part in a small trial of an experimental drug to treat the rare, degenerative disease with which he was born. A decade later, his memories of that time are fleeting. He remembers knowing the drug was intended to help his muscles to stay healthy despite the effects of Duchenne muscular dystrophy. He remembers being nervous about his first biopsy, having never undergone surgery before.…
Source: bizjournals.com Health Care:Physician Practices headlines - Category: American Health Authors: Source Type: news