At the initiative of Cure-SMA, the American association of patients suffering from SMN1-related proximal spinal muscular atrophy (SMA), experts have analysed all aspects of innovative treatments and have drawn up recommendations for good practice:
- these recommendations are aimed at healthcare professionals, patients themselves (and their families) and insurers,
- the authors have drawn on a wealth of literature on the subject to conduct a meta-analysis of current practices and the problems encountered,
- the urgency of treatment is reiterated, especially for the youngest patients and those most at risk of developing a severe form of the disease,
- indications in adults must always be carefully considered, with clear objectives,
- the conditions and procedures for switching between drugs were explained, with the need to allow time (6 to 12 months minimum) before doing so,
- information for families on tolerance problems and the risks incurred by gene therapy remains a topical issue.
Although this work should be seen in the context of the United States, where private insurance plays a significant role in indications, many of these recommendations are universal.
