Solid Biosciences is dedicated to developing new therapies to improve and extend the lives of patients with Duchenne muscular dystrophy (Duchenne) with a goal of reaching the largest number of patients in the shortest amount of time.
We are currently conducting a clinical trial to assess the safety and efficacy of an investigational gene transfer therapy for Duchenne.
Making our investigational therapy available through compassionate use or early/expanded access (an “Expanded Access Program”) is a complex matter that we have considered carefully. It is important to note that providing early access to an investigational therapy can involve unknown risks, as well as an unfounded assumption of therapeutic benefit. Furthermore, an Expanded Access Program may make it harder to complete the studies necessary for the regulatory review and approval process that is ultimately the fastest path to making new therapies available to as many patients as possible.
Based on these considerations, an Expanded Access Program isn't currently being offered by the company. We will continue to re-evaluate this policy. In the meantime, we encourage patients to speak with their physicians about the possibility of enrolling in clinical trials in order to gain access to investigational therapies. If you have questions about Solid Biosciences' Expanded Access Policy, please contact community@solidbio.com.