The U.K. has expanded the referral criteria for the approved gene therapy Zolgensma to include children with spinal muscular atrophy (SMA) who weigh up to 18 kilograms (40 lbs), according to a press release from SMA UK.
The expansion to a higher weight category — 15 to 18 kgs (33 to 40 lbs) — was advanced by the National Multidisciplinary Team (NMDT) which, along with infusion centers in England, provide services for children in England and Wales. The NMDT brings together infusion center representatives and others to ensure joint and equitable decision-making about the delivery of medicines such as Zolgensma.
Initially, the NMDT invited referrals for children, 12 months or younger, who had never been treated (treatment naïve) or were untreated. This included those 6 months and younger who were pre-symptomatic, or newly diagnosed and untreated. Children 7 to 12 months who were newly diagnosed and untreated were also eligible for referrals.
Read more at: https://smanewstoday.com/news-posts/2022/02/02/uk-expands-zolgensma-referral-criteria-heavier-children/