Muscular Dystrophy UK and SMA UK were extremely disappointed to learn earlier this week that, despite the appeals we and others have made, the Joint Drugs and Therapeutics Committee (JDTC) at Cambridge University Hospitals’ Trust has decided not to recommend the use of risdiplam for treating spinal muscular atrophy (SMA) in adults (with type I and type 2 SMA) through the Early Access to Medicines Scheme (EAMS). The local neuromuscular service team would have been keen to support the implementation of the EAMS if the Committee had approved it.
We are working through various routes to urge the Trust to change its position and in the meantime to address how people living in this area of the country who meet the EAMS eligibility criteria can get access to the risdiplam EAMS.