Does it cure spinal muscular atrophy (SMA)?

Drugs.com

Official answer

by Drugs.com

Evrysdi does not cure spinal muscular atrophy (SMA), it just replaces a protein that is low or missing in newborns, children, or adults with the condition, improving muscle strength and allowing some relief from some of the symptoms of SMA. Evrysdi must be taken for a person’s lifetime; if Evrysdi is stopped the newborn, child, or adult’s SMA symptoms will progressively worsen at a faster rate.

How does Evrysdi work?

Evrysdi (risdiplam) works by targeting the SMN2 gene (survival motor neuron gene 2), causing it to make more functional SMN protein. This increases SMN protein levels throughout the central nervous system and body, helping to improve motor nerve and muscle function in children and adults with SMA.

Evrysdi was approved by the FDA on August 7, 2020) and is available as a liquid that is given by mouth or through a gastrostomy tube (g-tube) once a day after a meal. It may be used to treat all types of spinal muscular atrophy (SMA) in adults, children, and newborns. A label extension for Evrysdi in May, 2022 to include pre-symptomatic infants aged under 2 months (or newborns) allows healthcare providers to intervene as early as possible in treating babies with SMA.

Related medical questions

Disclaimer

Every effort has been made to ensure that the information provided by Drugslib.com is accurate, up-to-date, and complete, but no guarantee is made to that effect. Drug information contained herein may be time sensitive. Drugslib.com information has been compiled for use by healthcare practitioners and consumers in the United States and therefore Drugslib.com does not warrant that uses outside of the United States are appropriate, unless specifically indicated otherwise. Drugslib.com's drug information does not endorse drugs, diagnose patients or recommend therapy. Drugslib.com's drug information is an informational resource designed to assist licensed healthcare practitioners in caring for their patients and/or to serve consumers viewing this service as a supplement to, and not a substitute for, the expertise, skill, knowledge and judgment of healthcare practitioners.

The absence of a warning for a given drug or drug combination in no way should be construed to indicate that the drug or drug combination is safe, effective or appropriate for any given patient. Drugslib.com does not assume any responsibility for any aspect of healthcare administered with the aid of information Drugslib.com provides. The information contained herein is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. If you have questions about the drugs you are taking, check with your doctor, nurse or pharmacist.

Popular Keywords