Spot the signs of SMA early to take action

Children with spinal muscular atrophy (SMA) lose essential motor neurons cells every day that passes without treatment. Spotting the signs and symptoms early is critical to getting a diagnosis and starting treatment. Remember, you know your child better than anyone. If you feel something is not right, trust your instincts and immediately contact your child’s doctor for a referral to a pediatric neurologist. Your doctor will determine diagnosis based on the results of a genetic test.

Here is what to look for if you suspect SMA.

Download the Facts About SMA guide
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Spot the signs of SMA early to take action

Children with spinal muscular atrophy (SMA) lose essential motor neurons cells every day that passes without treatment. Spotting the signs and symptoms early is critical to getting a diagnosis and starting treatment. Remember, you know your child better than anyone. If you feel something is not right, trust your instincts and immediately contact your child’s doctor for a referral to a pediatric neurologist. Your doctor will determine diagnosis based on the results of a genetic test.

Here is what to look for if you suspect SMA.

Download the Facts About SMA guide
Icons of babies to indicate that SMA Type 1 is the most common type of SMA and affects about 6 of every 10 children with SMA

SMA Type 1 is the most common type of SMA and affects about 6 of every 10 children with SMA

SMA Type 1 signs and symptoms

SMA Type 1 is the most common type of SMA. It is severe, and signs and symptoms usually begin to appear at less than 6 months of age. SMA Type 1 is a life-threatening condition that needs immediate attention. Signs and symptoms may vary at first, which means caregivers may not see them right away.

Signs and symptoms may include:

  • A “floppy” baby, or hypotonia
  • Small or weak muscles
  • Difficulty breathing/belly breathing
  • Feeding issues, like choking or trouble swallowing
  • Weak sucking and labored breathing during feeding
  • Tongue fluttering
  • Bell-shaped chest (a result of muscle weakness)
  • Weak cough
  • Lack of reflexes
  • “Frog legs,” or the inability to kick while lying on back
  • Lack of motor development, like being unable to lift head or roll over
  • Inability to sit
  • Weak cry

SMA Type 2 signs and symptoms

Muscle weakness is very common and affects the ability to stand or sit without assistance between the ages of 6 and 18 months. Typically, these children first start losing abilities before 2 years of age.

Cure SMA resources to help you spot the signs of SMA

Developmental delays are a red flag and can be early signs of a serious medical issue such as SMA. Download a guide from Cure SMA if your child is aged 0 to 6 months old or 7 to 12 months old to help spot the signs of SMA. Each guide includes a checklist of signs and symptoms to use when speaking to your pediatrician about your concerns. In addition, watch this video from Cure SMA about how to spot the warning signs of SMA.

SMA Type 3 signs and symptoms

Symptoms can start after 18 months of age, usually in early childhood, and children lose many functions as they grow older, including climbing and walking.

SMA Type 4 signs and symptoms

Symptoms can start as early as 18 years of age but typically don’t begin until after 20 or 30 years of age. People with SMA Type 4 usually only experience mild muscle weakness.

Download PDF for Facts About SMA

Does my child have SMA?

The Facts About SMA provides you with easy-to-understand information about what causes SMA, signs and symptoms of SMA, and how quickly SMA can progress to a life-threatening condition. Learn more about SMA and bring this PDF to your doctor’s office to determine next steps.

Download the facts

Important Safety Information

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

  • ZOLGENSMA can increase liver enzyme levels and cause acute serious liver injury or acute liver failure.
  • 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?

  • ZOLGENSMA can increase liver enzyme levels and cause acute serious liver injury or acute liver failure.
  • 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, if the patient misses a dose of corticosteroid or vomits it up, or if the patient experiences a decrease in alertness.

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

  • Infections before or after ZOLGENSMA infusion can lead to more serious complications. Contact the patient’s doctor immediately if you see any signs of a possible 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 the patient experiences unexpected bleeding or bruising.
  • Thrombotic microangiopathy (TMA) has been reported to occur approximately one week after ZOLGENSMA infusion. Caregivers should seek immediate medical attention if the patient experiences any signs or symptoms of TMA, such as unexpected bruising or bleeding, seizures, or decreased urine output.

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 a 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 Novartis Gene Therapies, Inc. at 833-828-3947.

Please see the Full Prescribing Information.