Family stories: Our experience with ZOLGENSMA

Hear from families living with spinal muscular atrophy (SMA) Type 1 as they share what the day of treatment with ZOLGENSMA® (onasemnogene abeparvovec-xioi) means to them and how their child is doing after treatment. Each story is unique in its journey toward treatment, but what they all have in common is a sense of hope and determination that will inspire you.

Family stories: Our experience with ZOLGENSMA

Hear from families living with spinal muscular atrophy (SMA) Type 1 as they share what the day of treatment with ZOLGENSMA® (onasemnogene abeparvovec-xioi) means to them and how their child is doing after treatment. Each story is unique in its journey toward treatment, but what they all have in common is a sense of hope and determination that will inspire you.

The children in these videos were diagnosed with SMA Type 1 at a time when there were no FDA-approved treatments for SMA. These children participated in group 2 of the START clinical study that evaluated ZOLGENSMA.

Results and outcomes vary among children based on several factors, including how far their SMA symptoms had progressed prior to receiving treatment.


FAMILY STORIES

See how ZOLGENSMA is changing spinal muscular atrophy (SMA) Type 1 treatment

Read the transcript

Lorem ipsum dolor sit amet, consectetur adipiscing elit. Praesent rhoncus vestibulum quam, at euismod diam consequat vitae. Suspendisse potenti. Integer aliquet, nibh sit amet viverra volutpat, est massa faucibus quam, et lobortis mi libero in ligula. Maecenas scelerisque diam nec tincidunt mattis. Nunc nunc ante, scelerisque sit amet massa at, accumsan molestie nisi. Mauris vel purus eget leo pharetra vestibulum. Nullam nisl dui, lobortis at porta eu, efficitur eget eros.

Donec pellentesque tincidunt mi, in ultrices massa dignissim eget. Maecenas quis eros imperdiet, condimentum lorem vitae, rutrum nisi. Suspendisse sollicitudin ante in nulla elementum, nec cursus orci tincidunt. Curabitur eget dolor et magna venenatis varius. Morbi mi ex, mollis lacinia consequat sed, fermentum vitae orci. Mauris semper tincidunt nisl, id sodales risus consequat sed. Nam a enim est. Nulla quis orci ut nisl faucibus commodo.

In hac habitasse platea dictumst. Orci varius natoque penatibus et magnis dis parturient montes, nascetur ridiculus mus. Curabitur lobortis venenatis scelerisque. Aliquam lacinia ornare diam non feugiat. Vivamus nec lorem ornare nibh lacinia rhoncus vitae ut arcu. Etiam aliquam, libero at rutrum volutpat, ante dui viverra neque, quis consectetur nibh orci id augue. Aenean vel sapien mauris. Cras ultricies efficitur magna nec volutpat. Maecenas porttitor mauris a consectetur scelerisque. Vestibulum eget neque sem. Pellentesque ipsum lorem, tempus in arcu sed, lacinia sodales tortor. Aenean pellentesque, felis in mollis fermentum, augue ligula hendrerit leo, pharetra finibus tortor nulla id nulla. In hac habitasse platea dictumst. Aenean ornare venenatis magna, non sollicitudin magna pretium a.

Nunc ut felis commodo, lacinia lorem eu, rhoncus lorem. Pellentesque vulputate consectetur lacus nec condimentum. Suspendisse bibendum nisl in ligula placerat viverra. Sed laoreet bibendum metus sed tristique. Quisque eleifend dui enim, sed mollis nisi maximus at. Curabitur molestie sagittis mauris, non porta lacus efficitur id. Aenean convallis convallis tortor sit amet rutrum. Vestibulum bibendum vitae sapien ac tempus. Phasellus eget consequat nulla. Nulla ullamcorper sodales semper. Suspendisse ex turpis, efficitur vitae sem vitae, pulvinar dictum arcu. Nullam iaculis ligula ut velit fermentum congue. Etiam luctus interdum condimentum. Aliquam rutrum, ligula sed luctus ornare, ipsum turpis consectetur justo, quis tristique turpis magna a erat. Mauris commodo luctus nulla et elementum.

Pellentesque dui ipsum, posuere vel felis vitae, vehicula tincidunt tortor. Donec tincidunt lectus metus, in hendrerit quam tempus eu. Sed et nibh vehicula, aliquam lorem ac, fermentum massa. Nam vel diam porttitor, tempor augue sed, pellentesque ligula. Ut gravida, risus in pretium suscipit, elit sem sagittis lectus, vitae pulvinar diam turpis nec felis. Phasellus congue justo sodales diam mattis ultrices. Integer sed varius purus. Integer vitae arcu urna.

Close the transcript
Play Button

NOW PLAYING

Adalyne’s story

When Patti and Brandon’s first daughter was diagnosed, they knew nothing about SMA. Hear how their journey was different with Adalyne, who was treated at about 2½ months old and is 3 years old in the video.

Watch the video
Play Button

NOW PLAYING

Donovan’s story

Meet Laura and Matt and their son, Donovan, who has SMA Type 1 and is 2½ years old in the video. He was treated with ZOLGENSMA at about 2 months of age. Learn how they found out about a clinical study and what Donovan can proudly accomplish.

Watch the video
Play Button

NOW PLAYING

Eli’s story

Natalie was in shock when her son, Eli, was diagnosed with SMA Type 1. Hear about the day Eli was treated when he was about 4 months old, the exciting moment when Natalie realized treatment was working, and what he can do as a 3½ year old in the video.

Watch the video
Play Button

NOW PLAYING

Evelyn’s story

Elena and Milan’s experience with SMA started even before their second daughter, Evelyn, was born with SMA Type 1. Hear about their incredible journey with Evelyn, who was treated at about 2 months old and is 3½ years old in the video.

Watch the video
Play Button

NOW PLAYING

Malachi’s story

Hear from Tina and Torence, whose son, Malachi, has SMA Type 1. Their story begins with heartbreak and ends with hope and joy for Malachi, who was treated at about 4 months old and is 2½ years old in the video.

Watch the video
Play Button

NOW PLAYING

Matteo’s story

Hear from Nicole and Derwin, whose son, Matteo, has SMA Type 1 and was treated at about 1 month old and is 3 years old in the video. Learn what a one-time-only treatment means to them, and see all the things Matteo loves to do.

Watch the video

After Malachi was treated, we were excited to see what would happen next.

Torence, father of Malachi

After Malachi was treated, we were excited to see what would happen next.

Torence, father of Malachi

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.