List of Treatments for Spinal Muscular Atrophy

Clinical trials

Overview
Depending on several factors, you may be eligible to participate in clinical trials studying new treatments for spinal muscular atrophy (SMA). Eligibility may be based on specifics of your or your child’s… read more

Evrysdi (Risdiplam)

Overview

Evrysdi is a prescription medication approved by the U.S. Food and Drug Administration (FDA) to treat people with spinal muscular atrophy (SMA) ages 2 months and older. Evrysdi is also known by its drug name,… read more

Exercise

Overview
Exercise can help most people stay healthy and feel their best. Exercise can reduce stress, lower blood pressure and cholesterol, improve sleep, promote a healthy weight, and help prevent the development of… read more

Gastrostomy tube (G-tube)

Overview
The gastrostomy tube (G-tube) is a flexible feeding tube surgically inserted through the abdominal wall and directly into the stomach. It bypasses the mouth and esophagus, delivering food, liquid, and… read more

Immunizations

Overview
Children or adults with SMA are at greater risk for pulmonary infection or difficulty breathing or clearing the lungs.¹ Therefore, people with SMA are at greater risk for serious complications and infections… read more

Invasive breathing support

Overview
If noninvasive ventilation (NIV) does not provide enough respiratory support, people with respiratory issues due to spinal muscular atrophy (SMA) may need greater assistance breathing.

With invasive… read more

Nasal feeding tubes

Overview
Nasal feeding tubes are temporary, non-surgical tubes placed through the nose and into the stomach or intestine. They provide short-term nutrition for people with spinal muscular atrophy (SMA) who have… read more

Nissen Fundoplication and G-Tube

Overview
Nissen fundoplication (NF) is a surgical procedure that involves wrapping the stomach around itself to prevent acid reflux. “Wrapping” tightens the sphincter muscle between the stomach and esophagus, making it… read more

Noninvasive breathing support

Overview
Some people with spinal muscular atrophy (SMA) require breathing assistance. The type of ventilation equipment or device used depends on the severity of the SMA symptoms.

Noninvasive ventilation is one type… read more

Occupational Therapy

Overview
Occupational therapy (OT) can help people with spinal muscular atrophy (SMA) increase independence in daily activities at home, work and in school. An occupational therapist plays an essential role in helping… read more

Physical therapy

Overview
Physical therapy (PT) can help adults and children with spinal muscular atrophy (SMA) maximize their function, strength, and endurance. Physical therapists (PTs) design treatments to promote movement, prevent… read more

Respiratory therapy

Overview
Weak respiratory muscles can make it difficult for people with spinal muscular atrophy (SMA) to cough and clear mucus from their lungs, which puts them at higher risk for severe respiratory infections and… read more

Speech and language therapy

Overview
A speech-language pathologist (SLP) can help people with spinal muscular atrophy (SMA) overcome problems with speech and swallowing that develop due to weakness in the tongue, throat, and respiratory muscles.… read more

Spinraza (Nusinersen)

Overview
Spinraza is a prescription medication approved by the U.S. Food and Drug Administration (FDA) for treating spinal muscular atrophy (SMA) in children and adults. Spinraza is also known by its drug name,… read more

Surgery for scoliosis

Overview
About 90 percent of children with spinal muscular atrophy (SMA) develop scoliosis, an abnormal curvature of the spine. Scoliosis can interfere with movement, make breathing more difficult, and worsen posture.… read more

Vertical expandable prosthetic titanium rib (VEPTR)

Overview
The vertical expandable prosthetic titanium rib (VEPTR) is an invasive breathing support device approved by the U.S. Food & Drug Administration (FDA) to treat children with thoracic insufficiency syndrome (TIS)… read more

Zolgensma (Onasemnogene abeparvovec-xioi)

Overview
Zolgensma is a prescription medication approved by the U.S. Food and Drug Administration (FDA) for treatment of spinal muscular atrophy (SMA) in children under two years of age. Zolgensma is also known by its… read more