Expanded access program with nusinersen in SMA type i in italy: strengths and pitfalls of a successful experience


We would like to share our experience with the expanded access program (EAP) using Nusinersen in infants and children with type 1 Spinal muscular atrophy (SMA). EAP is the use of an investigational new drug outside of a clinical trial in patients for the diagnosis, monitoring, or treatment of a serious disease or condition. EAP provides a pathway for patients to gain access to investigational drugs, biologics, and medical devices used to diagnose, monitor, or treat patients with serious diseases or conditions for which there are no comparable or satisfactory therapy options available outside of clinical trials.


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