This article is sponsored by Genentech, Inc.
Spinal muscular atrophy (SMA) can have a widespread impact, including muscle atrophy, weakness, and related conditions that worsen over time. Join Shadé Moody, MD, and SMA community members as they share their perspectives on the SMA journey and discuss an approved treatment.
SMA, a rare genetic disease, is caused by insufficient levels of survival motor neuron (SMN) protein throughout the body.1 SMN protein deficiencies can have a widespread impact, including motor neuron degeneration that leads to muscle atrophy, weakness, and related conditions that worsen over time.2,3
In later-onset (Type 2 or 3) SMA, patients experience motor function decline that can begin in childhood and continue throughout their lifespan.4-8 In infantile-onset (Type 1) SMA, most patients are not expected to survive beyond 2 years.9 In fact, no more than approximately 25% of patients are expected to survive longer than 14 months without permanent ventilation.9 Understanding the natural history of SMA helps healthcare providers, patients, and caregivers establish appropriate treatment goals.
Join Shadé Moody, MD, and members of the SMA community to hear their perspectives on the SMA journey, including the role of Evrysdi® (risdiplam) in the treatment of infantile- and later-onset SMA in patients 2 months of age and older.10 Please see Important Safety Information below.
Evrysdi is a once-daily oral SMN2-splicing modifier that is designed to address the underlying cause of SMA.10 The Evrysdi clinical trial program reflects the real-world population and includes more than 450 patients with SMA who have a broad range of disease severity and functional ability.10
Explore the results of the Evrysdi clinical trial program and review the efficacy and safety data in the SMA population in this upcoming National Broadcast featuring Dr Moody and SMA community members.
Register now for your preferred session. Available times:
- January 25, 2022, at 6:00 PM ET
- January 26, 2022, at 8:00 PM ET
There will be an opportunity to ask questions during the faculty Q&A portion of the upcoming National Broadcast.
Please note that this is a promotional education program; CME credit will not be available.
Evrysdi® (risdiplam) is indicated for the treatment of spinal muscular atrophy (SMA) in patients 2 months of age and older.
Important Safety Information
Interactions With Substrates of MATE Transporters
- Based on in vitro data, Evrysdi may increase plasma concentrations of drugs eliminated via MATE1 or MATE2-K, such as metformin
- Avoid coadministration of Evrysdi with MATE (multidrug and toxin extrusion) substrates. If coadministration cannot be avoided, monitor for drug-related toxicities and consider dosage reduction of the coadministered drug if needed
Pregnancy & Breastfeeding
- Evrysdi may cause embryofetal harm when administered to a pregnant woman. In animal studies, administration of Evrysdi during pregnancy and/or lactation resulted in adverse effects on development. Advise pregnant women of the potential risk to the fetus
- Pregnancy testing is recommended prior to initiating Evrysdi. Advise female patients to use contraception during treatment with Evrysdi and for at least 1 month after the last dose
- The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for Evrysdi and any potential adverse effects on the breastfed infant
Potential Effects on Male Fertility
- Counsel male patients that fertility may be compromised by treatment with Evrysdi. Male patients may consider sperm preservation prior to treatment
Most Common Adverse Reactions
- The most common adverse reactions in later-onset SMA (incidence in at least 10% of patients treated with Evrysdi and more frequent than control) were fever, diarrhea, and rash
- The most common adverse reactions in infantile-onset SMA were similar to those observed in later-onset SMA patients. Additionally, adverse reactions with an incidence of at least 10% were upper respiratory tract infection, pneumonia, constipation, and vomiting
You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.
Please see full Prescribing Information for additional Important Safety Information.
1. Lefebvre S, Burlet P, Liu Q, et al. Correlation between severity and SMN protein level in spinal muscular atrophy. Nat Genet. 1997;16(3):265-269.
2. Singh RN, Howell MD, Ottesen EW, Singh NN. Diverse role of survival motor neuron protein. Biochim Biophys Acta Gene Regul Mech. 2017;1860(3):299-315.
3. D’Amico A, Mercuri E, Tiziano FD, Bertini E. Spinal muscular atrophy. Orphanet J Rare Dis. 2011;6:71.
4. Pera MC, Coratti G, Mazzone ES, et al. Revised upper limb module for spinal muscular atrophy: 12 month changes. Muscle Nerve. 2019;59(4):426-430.
5. Mercuri E, Lucibello S, Pera MC, et al. Long-term progression in type II spinal muscular atrophy: a retrospective observational study. Neurology. 2019;93(13):e1241-e1247.
6. Coratti G, Pera MC, Lucibello S, et al. Age and baseline values predict 12 and 24-month functional changes in type 2 SMA. Neuromuscul Disord. 2020;30(9):756-764.
7. Coratti G, Lucibello S, Pera MC, et al. Gain and loss of abilities in type II SMA: a 12-month natural history study. Neuromuscul Disord. 2020;30(9):765-771.
8. Coratti G, Messina S, Lucibello S, et al. Clinical variability in spinal muscular atrophy type III. Ann Neurol. 2020;88(6):1109-1117.
9. Finkel RS, McDermott MP, Kaufmann P, et al. Observational study of spinal muscular atrophy type 1 and implications for clinical trials. Neurology. 2014;83(9):810-817.
10. Evrysdi [package insert]. South San Francisco, CA: Genentech, Inc.; 2021.
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