Spinraza (), by Biogen, was the first disease-modifying therapy for SMA that the FDA approved in December 2016 to treat SMA in both infants and adults.The treatment also boosts the ability of the SMN2 gene to produce more functional SMN protein.. Zolgensma. Roche’s Risdiplam Shows Significant Improvement in Survival and Motor Milestones in Infants With Type 1 Spinal Muscular Atrophy (SMA). If your child meets these criteria, we will complete a clinical evaluation and additional testing to determine if they are a candidate to receive gene therapy for SMA. Spinal Muscular Atrophy: What You Need to Know, Spinal Muscular Atrophy: Types and Symptoms, Spinal Muscular Atrophy: Testing and Diagnosis, spinraza-hcp.com/content/dam/commercial/spinraza/hcp/en_us/pdf/spinraza-prescribing-information.pdf, avexis.com/content/pdf/prescribing_information.pdf, roche.com/media/releases/med-cor-2020-04-28.htm, https://www.novartis.com/news/media-releases/zolgensma-data-shows-rapid-significant-clinically-meaningful-benefit-sma-including-prolonged-event-free-survival-motor-milestone-achievement-and-durability-now, roche.com/dam/jcr:29942591-7289-42f7-88a8-99da7f734952/en/roche-media-release-06022020-en.pdf, roche.com/dam/jcr:565ae565-4184-4aaf-8270-b368c5f65e2f/en/roche-mediarelease-risdiplam-07042020.pdf, ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Spinal-Muscular-Atrophy-Fact-Sheet, nejm.org/doi/pdf/10.1056/NEJMoa1702752?articleTools=true, n.neurology.org/content/94/15_Supplement/2493, sciencedirect.com/science/article/pii/S0960896617312841, SMA is caused by defects (mutations or deletions) in the. Children receive only one dose of gene therapy for SMA. Now that there is treatment available, the federal government in July added SMA to its official list of recommendations for newborn screening. SMA is a genetic disease that affects the spinal cord and nerves, resulting in muscle wasting and weakness. Gene replacement therapy for SMA is called onasemnogene abeparvovec-xioi (brand name Zolgensma). Treatment was also found to be safe and well-tolerated. Gene replacement therapy for SMA is called onasemnogene abeparvovec-xioi (brand name Zolgensma). Céline’s symptoms dramatically improved after she received a new gene therapy treatment for Type 1 spinal muscular atrophy at CHOP. To understand how the new treatments work, here is a quick reminder of what causes SMA: Nusinersen was approved in 2016 for all types of SMA. Roche provides regulatory update on risdiplam for the treatment of spinal muscular atrophy (SMA). Even better results have been found in babies treated before they have symptoms, she adds.4 Researchers are also testing whether gene therapy is beneficial when given in older children (up to age 5) age who can sit but not stand or walk. Roche. Use birth control while on treatment and for at least 1 month after stopping Evrysdi, are an adult male planning to have children: Evrysdi may affect a man’s ability to have children (fertility). “Onasemnogene is very effective in symptomatic infants who are treated early in their disease,” Dr. Chiriboga says. ©2020 The Children’s Hospital of Philadelphia. (2020. You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. Importantly, nearly all (about 95%) of the children maintained their ability to swallow, and 90% of them were able to feed orally, which is really striking,” Dr. Chiriboga says.3, A second trial found benefit after 1 year of treatment in patients 2 to 25 years of age with SMA type 2 or 3 compared with placebo.5 As expected, the benefits were greatest in younger people; however, disease stabilization (symptoms did not worsen) was found in people ages 18 to 25 years. If you are pregnant, or are planning to become pregnant, ask your healthcare provider for advice before taking this medicine. This section provides more information on these treatments as well as supportive therapies, but as always, talk to your doctor to see which treatment is right for you or your child. Children’s Hospital of Philadelphia has been involved in gene therapy clinical trials for SMA since the preliminary stages, gaining early clinical experience administering this new treatment. ), Roche. Share your story. In addition to treatment from a neurologist, these centers provide genetic counseling, physical/occupational therapy, pulmonology, orthopedic care, respiratory care, and nutritional care. It is not known if Evrysdi passes into breast milk and may harm your baby. If you do not live near a neuromuscular center, find a neuromuscular specialist or neurologist experienced in SMA who can begin treatment immediately. Please see full Prescribing Information for additional Important Safety Information. All generally result in worsening muscle weakness associated with muscle twitching. Nusinersen was approved by the US Food and Drug Administration (FDA) based on findings from a clinical trial in which 51% of babies with symptoms of SMA type 1 treated with this drug showed significant improvement in motor function, including: …compared with none of the babies given placebo (no drug). Without enough SMN proteins, motor neurons die and muscles become weak. Spinal muscular atrophy (SMA) is a group of neuromuscular disorders that result in the loss of motor neurons and progressive muscle wasting. Risdiplam also is being studied in people with SMA ages 6 months to 60 years who previously used other SMA treatments.6, The most common side effects of risdiplam in these trials were upper respiratory tract infection, pneumonia, fever, constipation, and cold symptoms.5,6 In some cases, pneumonia can be severe, and in rare cases, people developed lung infection, respiratory failure, and decreased muscle tone.5,6. “Before December 2016, there was nothing on the horizon for SMA treatment. ), Novartis. CHOP-INTEND, a measure of motor skills for SMA type 1 infants, works on a 0 to 64-point scale with higher scores indicating better motor function.

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