South African clinicians have compiled and analysed the difficulties in accessing innovative treatments for SMN1-related proximal spinal muscular atrophy in much of the non-Western world:
- their work is based on an exhaustive literature review covering the period 2014–2025, though limited to the paediatric population, innovative treatments (Spinraza, Zolgensma and Evrysdi) and their corollary, neonatal screening;
- a glaring inequality of access is reported in the 40 articles selected for the study, raising ethical questions;
- a lack of resources, the disorganisation of healthcare systems and the lack of long-term data on the efficacy and tolerability of these otherwise very expensive drugs constitute major barriers to making these therapies available to as many people as possible.
The authors call for concerted efforts among the various stakeholders at a global level to combat this injustice.
