Spinal Muscular Atrophy
Treatment

Children with spinal muscular atrophy (SMA) are treated at our Pediatric Muscular Dystrophy Center. They require ongoing, specialized care from experts, including neurologists, orthopedists, pulmonologists and surgeons. Although there is no cure for the disease, its symptoms and complications can be successfully managed.

Several medications are being studied for this disease. Treatments may include:

Medications

Riluzole blocks certain proteins in the central nervous system that may cause damage to nerve cells. This drug is prescribed for adults with amyotrophic lateral sclerosis (ALS), also called Lou Gerhig's disease, which is similar to SMA. Preliminary studies have suggested a benefit for some children with SMA, but doctors believe that more studies must be completed before the benefits for children are confirmed.

Hydroxyurea, a chemotherapeutic agent, and valproic acid, also known by the brand name Depakote, are being studied for possible benefits in children with SMA. Due to potentially serious side effects, it is recommended that you discuss these medications with a specialist before you give them to your child.

Nutrition

Children with type I and some with type II SMA have special nutritional needs because of chewing and swallowing problems. Special X-ray studies, called a swallowing study and cine esophagram, may help determine the foods that are dangerous to your child.

Our therapists will work with you and your child to ensure your child receives adequate nutrition. Children who are unable to swallow liquids and semi-solids may need a gastrostomy tube, which allows nourishing liquids to flow directly to the stomach. These tubes are inserted by a surgeon or gastroenterologist.

Physical Therapy

Muscle weakness in the legs and arms may cause a child to experience tightness in the joints, called contractures. Our physical therapists can teach your child special range-of-motion exercises to keep muscles flexible and mobile. Night splints on ankles and wrist also may help prevent contractures or joint tightness.

Mobility Aids

Children with type I and type II SMA typically are unable to stand or walk independently and require an electric wheelchair. Many children can safely operate a wheelchair by age 2 or 3.

Respiratory Support

Children with SMA have a high risk of developing respiratory problems. Many respiratory support therapies are recommended to help prevent complications. Treatments may include:

  • Breathing exercises.
  • Breathing devices when breathing muscles don't function properly. Devices such as "negative pressure" ventilators and external positive airway pressure support systems can help. Positive airway systems use a gently placed (but snug) mask, to deliver air directly to the nostrils.
  • Supplemental oxygen, usually delivered through a small nasal tube.
  • Yearly immunizations to avoid preventable illnesses, such as influenza.

Reviewed by health care specialists at UCSF Benioff Children's Hospital.

Related Information

UCSF Clinics & Centers

Pediatric Brain Center

Neuromuscular Clinic
1825 Fourth St., Sixth Floor
San Francisco, CA 94158
Phone: (415) 353-7596
Fax: (415) 353-2400
Appointment information