Spinal muscular atrophy (SMA) is a complex neuromuscular disorder requiring a comprehensive, multidisciplinary approach to optimize patient outcomes. While advances in disease-modifying therapies (DMTs) have transformed treatment, these therapies alone are not enough. A collaborative team of specialists is essential to address the diverse medical, rehabilitative, and psychosocial needs of patients with SMA throughout their lives.

Multidisciplinary care is critical in treating patients with SMA. One of the foundations of the SMA care guidelines is multidisciplinary care, which is more important than ever for the treatment of patients with SMA. A key component of the care team that is frequently left out is the primary care physician (PCP). In our clinic, as soon as a patient is diagnosed with SMA, we send a copy of the most updated version of the care guidelines and considerations to the patient’s PCP, who is an anchor for the patient and for the rest of the team.

The care team for patients with SMA truly needs to include a variety of subspecialists, as it is difficult for a single provider to manage all of the issues involved. The pulmonologist, orthopedist, neurologist, physiatrist, and physical and occupational therapists are vital in the care of patients and in helping them to live their best possible lives. Genetic counselors are often the initial point of contact with PCPs and with patients in certain states when the newborn screening results are positive. In addition, endocrinologists and nutritionists are vital in terms of helping patients maintain bone health, optimal growth, and nutrition. Older patients may also need obstetrics and gynecology practitioners, urologists, social workers, and mental health providers as part of the team. Finally, one of the most important people in a multidisciplinary care team is the care coordinator, often a nurse or nurse practitioner, who helps to coordinate all the team members. And at the very center of the team and its focus are the patient and their family.

In the last several years, the multidisciplinary care team has become more critical, not less critical. There is a misconception that, because we have DMTs and we are able to treat newborn patients, we do not have a need for the multidisciplinary team. I think there is nothing that could be further from the truth. For instance, we are now seeing neurocognitive issues that were previously unseen in some children who have more severe SMA, and we may need to involve neuropsychologists in the care of these patients. With the early use of DMTs, some children who were not going to walk are now walking. However, over time, they may begin to have difficulty with ambulation and early scoliosis, and it requires a team to identify this and to learn how to care for these patients. We have to be prepared with an infrastructure to care for our patients with SMA if adverse events arise with novel therapies. Although we may add new team members, I do not think that any team members who are currently in place should leave the team.