Background Risdiplam is the first oral spinal muscular atrophy (SMA) treatment and was made available in the UK through an Early Access to Medicines Scheme (EAMS), which enrolled patients between September 2020–July 2021.

Methods Risdiplam’s EAMS indication was for patients with Type 1/2 SMA, ≥2 months old and not suitable for authorised SMA treatments. Patient characteristics and risdiplam-provision data from treating physi- cians, and patient/caregiver feedback, were collected.

Results Of 242 patients enrolled, 92 were children (<18 years, 38.0%) and 150 were adults (62.0%). Most had Type 2 SMA (90.1%; Type 1: 9.9%). Most commonly reported comorbidities and clinical management characteristics were scoliosis (99/242; 40.9%), previous spinal surgery (82/242; 33.9%) and ventilatory support (79/242; 32.6%). Risdiplam was the first disease-modifying SMA therapy that most patients had received (155/242; 64.0%), including 7 (aged 15–33) with the more severe Type 1 form. Most patients who received risdiplam did so through sponsor-funded home delivery vs. hospital pharmacy collection (226/234; 96.6% vs. 6/234; 2.6% [not reported: 2/234]).

Conclusions Risdiplam was made available to a varied SMA population through the EAMS, targeting patients otherwise unable to access disease-modifying treatment. Many patients received disease- modifying treatment for the first time, addressing a significant unmet need.