I’ll always remember the day

we received the one-time-only dose for SMA

See how ZOLGENSMA works

ZOLGENSMA targets the genetic root cause of SMA

ZOLGENSMA (pronounced zol-JEN-smuh) replaces the function of the missing or nonworking survival motor neuron 1 (SMN1) gene with a new, working copy of a human SMN gene that helps motor neuron cells work properly.

Watch the video
Evelyn, who has SMA Type 1, standing by a window
Malachi, who has SMA Type 1, laughing while he holds a dandelion
Donovan, who has SMA Type 1, holding a blue toy

Twelve patients with SMA Type 1 took part in group 2 of the START clinical study of ZOLGENSMA. Listen to some of the families share their SMA journey, hear about the day their child was given ZOLGENSMA, and learn how their child is doing after treatment.

Hear their stories
Evelyn, who has SMA Type 1, standing by a window

Evelyn, treated at
~2 months and pictured at 3½ years,
has SMA Type 1.

Watch Evelyn’s Video
Malachi, who has SMA Type 1, laughing while he holds a dandelion

Malachi, treated at
~4 months and pictured at 2½ years,
has SMA Type 1.

Watch Malachi’s Video
Donovan, who has SMA Type 1, holding a blue toy

Donovan, treated at
~2 months and pictured at 2½ years,
has SMA Type 1.

Watch Donovan’s Video
Remember the day - ZOLGENSMA® (onasemnogene abeparvovec-xioi) calendar

ZOLGENSMA is a one-time-only dose

ZOLGENSMA is given intravenously (IV) over the course of 60 minutes.

Learn more
The OneGene Program™

Support you can count on

The OneGene Program™ is a team of highly trained and dedicated people who help ensure that every family has the support they need during their treatment journey and beyond.

Meet the team

Adalyne, treated at ~2½ months and pictured at 3 years, has SMA Type 1.

A doctor with a stethoscope

Getting started on ZOLGENSMA

Know what to expect before, during, and after treatment.

Start now
A baby with a pacifier

Results that make a difference

Learn about the results of 2 clinical studies.

View study results
A video with a play button

Our experiences with ZOLGENSMA

Listen to families talk about the day their child was given ZOLGENSMA and how their child is doing after treatment.

Watch the videos

Important Safety Information

What is the most important information I should know about ZOLGENSMA?

  • Liver enzymes could become elevated and cause acute serious liver injury in children who receive ZOLGENSMA.
  • Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.

Important Safety Information

What is the most important information I should know about ZOLGENSMA?

  • Liver enzymes could become elevated and cause acute serious liver injury in children who receive ZOLGENSMA.
  • Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.
  • Contact the patient’s doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish, or if the patient misses a dose of the corticosteroid or vomits it up.

What should I watch for before and after infusion with ZOLGENSMA?

  • Viral respiratory infections before or after ZOLGENSMA infusion can lead to more serious complications. Contact the patient’s doctor immediately if you see signs of a possible viral respiratory infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.
  • Decreased platelet counts could occur following infusion with ZOLGENSMA. Seek immediate medical attention if a patient experiences unexpected bleeding or bruising.

What do I need to know about vaccinations and ZOLGENSMA?

  • Talk with the patient’s doctor to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid.
  • Protection against respiratory syncytial virus (RSV) is recommended.

Do I need to take precautions with the patient’s bodily waste?
Temporarily, small amounts of ZOLGENSMA may be found in the patient’s stool. Use good hand hygiene when coming into direct contact with bodily waste for 1 month after infusion with ZOLGENSMA. Disposable diapers should be sealed in disposable trash bags and thrown out with regular trash.

What are the possible or likely side effects of ZOLGENSMA?
The most common side effects that occurred in patients treated with ZOLGENSMA were elevated liver enzymes and vomiting.

Indication

What is ZOLGENSMA?
ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is given as a one-time infusion into the vein. ZOLGENSMA was not evaluated in patients with advanced SMA.

The safety information provided here is not comprehensive. Talk to the patient’s doctor about any side effects that bother the patient or that don’t go away.

You are encouraged to report suspected side effects by contacting the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch, or AveXis at 833-828-3947.

Please see the Full Prescribing Information.