Spinal muscular atrophy (SMA) is a rare autosomal recessive neuromuscular disorder caused by biallelic variants in the SMN1 gene. The treatment setting for SMA has expanded significantly since the approval of nusinersen (Spinraza; Biogen) in December 2016, with two additional SMN-enhancing therapies approved, including onasemnogene abeparvovec (Zolgensma; Novartis) and risdiplam (Evrysdi; Roche).
The expansion of treatment options has changed what clinicians can offer to patients with SMA. However, it has not resolved the issue of where patients receive care. Coordinated multidisciplinary management has long been identified as central to treatment in SMA, spanning physical and occupational therapy, respiratory care, nutrition, speech-language pathology, and psychosocial support.
Treatment options for SMA continue to grow. A tablet formulation of risdiplam was approved in February 2025, an intrathecal formulation of onasemnogene abeparvovec (Itvisma) was approved in November 2025, and a high dose regimen of nusinersen was approved in March 2026. Additionally, a decision on apitegromab (Scholar Rock), an investigational myostatin inhibitor for SMA, by the FDA is expected by September 30, 2026.
Assembling and sustaining a multidisciplinary care team, particularly outside academic centers, remains a practical barrier. This determines whether patients reach the clinicians equipped to treat them. The report highlights that this is an organizational problem as much as a clinical one.
A multidisciplinary care team for SMA typically includes a physical therapist, an occupational therapist, respiratory therapists, a nutritionist, a speech therapist, and a social worker. The most effective care can be rendered by this multidisciplinary approach, where a patient comes for one visit and is evaluated by all the necessary disciplines, and the team puts their heads together and comes up with a unified plan of treatment.
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Sandeep Rana, MD, a neurophysiologist and director of the ALS Center at the Allegheny Health Network Neuroscience Institute, has established a multidisciplinary clinic for SMA. He notes that the arrival of disease-modifying therapies has shifted the team’s priorities toward wellness and long-term function.
Rana’s clinic has operated for close to 2 decades, initially established for amyotrophic lateral sclerosis (ALS) and later expanded to serve patients with SMA. Rana emphasizes the importance of coordinated multidisciplinary management in SMA treatment.
One challenge in coordinating care is the difficulty of staffing intrathecal administration. Rana notes that it is difficult to find a specialist who is well-trained and skilled at providing and doing the spinal tap and intrathecal administration of the medication.
Rana’s clinic has been successful in putting together a multidisciplinary team. He notes that the key is to get the word out and make clinicians aware of the effective medicines available and the centers that are providing the necessary care, including those focused on SMA awareness.
As the treatment setting for SMA continues to evolve, it is essential to prioritize coordinated multidisciplinary management and to address the challenges in coordinating care across different subspecialties. By doing so, clinicians can provide the best possible care for patients with SMA and help them to manage their condition effectively.