Spinal Muscular Atrophy Center

The Spinal Muscular Atrophy (SMA) Center is a multi-specialty clinic at The Johns Hopkins Hospital, specializing in diagnosis and treatment of SMA and related disorders. Our team shares decades of combined experience and includes experts in all aspects of SMA, such as clinical care, clinical trials and research. This experience gives us advantage in providing accurate diagnoses, especially for infants. The Johns Hopkins SMA Center has access to the most advanced treatments and is the only center in the country that provides continuous care for patients from infancy through adulthood.

About Spinal Muscular Atrophy

Spinal muscular atrophy is a disease that affects nerves in the spinal cord that control voluntary muscle movement. The muscles of the body cannot respond to signals from the nerves and begin to weaken and shrink (atrophy).

Our Mission

Patient Care

  • Provide expert, multi-specialty care to patients with SMA and related disorders, from infancy through adulthood
  • Administer new FDA-approved treatments
  • Offer support services to those living with SMA and their families


  • Participate in multicenter clinical trials of novel SMA therapeutics
  • Further understand the pathogenesis of SMA through integrating clinical and preclinical studies


  • Inspire and train the next generation of researchers and clinicians to address this devastating disorder
  • Increase public awareness and educate patients and their families about SMA

Life Saving Spinal Muscular Atrophy (SMA) Treatment | Sam's Story

Before a gene therapy medication was developed that changes the way the broken gene in SMA is expressed, there was no way to slow the progression of weakness. Thanks to a huge team effort at Johns Hopkins, this life-saving medication has been given over 500 times and has made a profound impact on patients’ lives. Meet the patient who received the 500th dose.

Gene Transfer Therapy for the Treatment of Rare Pediatric Neurologic Disorders

Pediatric neurologist Tom Crawford discusses how gene transfer therapy is being used to treat rare neurologic diseases in children, how it’s used at Johns Hopkins to treat infants diagnosed with SMA, and future challenges the field of gene transfer therapy must address.

Our Team

In addition to our core team of pediatric neurologists, we partner with others across Johns Hopkins, including specialists in orthopaedics, anesthesiology, genetics, gastroenterology and pulmonary medicine, to provide well-rounded care for children with SMA. Dr. Sumner and Dr. Crawford have also served as consultants to companies developing SMA treatment.

New Treatments for Spinal Muscular Atrophy Provide Hope for Many Neurodegenerative Diseases

The success and challenges of existing SMA treatments provide important insights for developing therapies for other neurological diseases. Charlotte Sumner, M.D., discusses research that helps identify new targets for these therapies.