EAN 2022 | Recommended standards of care for adult patients with spinal muscular atrophy

We still have the multidisciplinary care recommendations that have already included medication. They definitely stress the need of rehabilitation, physiotherapy for many patients, respiratory support, also addressing the nutrition, orthopedic care, different things depending on the current functional status and the age of the patient. We would treat differently or offer different kind of multidisciplinary care to those who are non-sitters, sitters, standers, or walkers. And this goes across all the ages of SMA patients. But medication plays a major role in the process.

But of course, to get the optimal result, we need to employ different parts of this multidisciplinary care, tailor them to the patient’s need. And this is also the biggest challenge, I believe, for the adult patients, because the pediatric patients have been followed up by pediatric centers since diagnosis. Adult patients come back to adult care centers. Often they are in their thirties or forties and have not been seen by a neurologist for a long time.

Access to treatment depends on the local regulations that may be different, different reimbursement strategies in different countries. But with two different medications that are now approved for SMA, some of it could also, let’s say, include decision making process done by the patient themselves, or let’s say, discussion of the best possible option for the patient from the point of view of the patient and the treating physician. We do not still have, let’s say, all the answers to the important questions that will come up during such discussions, but definitely the adults with SMA is the patient that needs to be treated.