Spinal Muscular Atrophy Program | Overview
Children with spinal muscular atrophy (SMA) need care from experts in several medical fields. The Spinal Muscular Atrophy Program at Boston Children’s Hospital brings together a team of specialists who are experienced in caring for children with this rare and complex condition.
Directed by Basil Darras, MD, our program brings together specialists from a variety of fields including:
- physical therapy
- social work
Our clinical teams are experienced in caring for children with SMA. For example, children with SMA can have complications when having anesthesia because of weakness in the respiratory muscles. Our team of anesthesiologists understands SMA and has the expertise to safely provide anesthesia for our SMA patients.
Leading the way in SMA treatment
In recent years, several new treatments have been FDA-approved for SMA, including gene replacement treatment and two drugs. As a leader in caring for children with SMA, we have been a part of the clinical trials for all of these treatment options and have extensive experience prescribing them for our patients.
A dedicated SMA clinic
We hold a dedicated SMA clinic once or twice a month. The clinic allows you to meet with all of your child’s SMA-related providers during a single visit. It also gives us the opportunity to evaluate your child’s health as a whole and to work with your family to make the best treatment decisions.
During clinic, our core team of physicians, specialists, and therapists sees patients together. When you come for an appointment during this clinic, your child will see several kinds of specialists:
- physical therapy
Our clinic also gives you the chance to meet other families affected by SMA and to learn about research opportunities.
We use a number of different tests to help us accurately diagnose your child and evaluate his or her condition. Our program takes advantage of several superb clinical and laboratory resources at Boston Children’s, including:
- Genetic testing, which confirms the diagnosis of SMA.
- Electromyography (EMG) and nerve conduction velocity (NCV) studies record the activity of muscles and motor and sensory nerves. Boston Children’s has its own EMG laboratory, which is staffed by the neurologists in our program.