Coverage Policy Manual
Policy #: 2025026
Category: Pharmacy
Initiated: November 2025
Last Review: September 2025
Onasemnogene abeparvovec-xioi (e.g., Zolgensma)
Description:
Spinal muscular atrophy (SMA) is an inherited disorder caused by homozygous deletions or variants in the SMN1 gene in chromosome 5. This gene is responsible for producing the “survival of motor neuron” protein (SMN1). Because of absent or low levels of survival motor neuron 1 protein, the motor neurons in spinal cord degenerate, resulting in atrophy of the voluntary muscles of the limbs and trunk. During early development, these muscles are necessary for crawling, walking, sitting up, and head control. The more severe types of SMA can also affect muscles involved in feeding, swallowing, and breathing. Treatments include:
1. Nusinersen (e.g., Spinraza) (see policy number 2019011) is a synthetic antisense oligonucleotide designed to bind to a specific sequence in exon 7 of the SMN2 transcript causing the inclusion of exon 7 in the SMN2 transcript, leading to the production of full length functional survival motor neuron 2 protein, which is very similar to SMN1.
2. Onasemnogene abeparvovec-xioi (e.g., Zolgensma) is an adeno-associated virus vector-based gene therapy and is intended as a one-time gene replacement therapy designed to deliver a functional copy of the SMN1 gene to motor neuron cells of individuals with spinal muscular atrophy. Because motor neurons are nondividing cells, it is postulated that once the SMN1 gene is incorporated in the cells, it would be retained over time and potentially allow for long-term, sustained survival motor neuron protein expression.
3. Risdiplam (e.g., Evrysdi) (see policy number 2025027) is a once-daily self-administered oral therapy. It is a selective SMN2 splicing modifier designed to bind with specificity to SMN2 pre-mRNA to modulate SMN2 pre-mRNA splicing towards the production of full-length SMN2 mRNA.
 
The exact role of the SMN protein in motoneurons has not been completely elucidated and levels of the SMN protein required for optimal functioning are unknown (Wang, 2007). SMN2 is a nearly identical modifying gene capable of producing nearly identical compensatory SMN2 protein. However, 70% to 90% of the transcripts produced from the SMN2 gene produce a truncated protein that is defective and unstable due to lack of exon 7 (Lorson, 1999). Further, humans exhibit variability (range, 0-6) in the number of copies of the SMN2 gene and copy number is inversely proportional to severity of disease (Lefebvre, 1997). These factors in tandem lead to wide variability in disease severity.
 
SMA is classified into 4 main categories (with additional subcategories) based on the age at the onset of symptoms (Muscular Dystrophy Association, 2017; National Organization for Rare Disorders, 2012). Generally, early onset of disease directly correlates to severity of symptom and rate of disease progression. There is no exact marker to classify these categories, and they are not well-distinguished by ICD-10-CM code.
 
    •  Type 0: The most severe form of SMA, symptoms can often be seen in the later stages of pregnancy. Fetal movements are less than expected and, after birth, the infant will have little ability to move and may not be able to breathe and swallow independently. Death occurs before the age of 6 months. SMN2 copy number 1.
    •  Type I (also called infantile SMA or Werdnig-Hoffman disease and subcategorized as IA, IB and IC): Onset within 6 months of birth and symptoms progress rapidly, and most infants die before 1 year of age from respiratory failure. About 60% of individuals with SMA constitute of this phenotype. SMN2 copy number 2. (Zerrres, 1995; Finkel, 2017).
    •  Type II (also called intermediate SMA or Dubowitz disease): Onset within 6 to 18 months with a less severe progression. Typically, a child can sit independently if positioned, but is unable to walk. More than 70% of individuals live beyond 25 years of age with adequate supportive care. SMN2 copy number 3 or 4.
    •  Type III (also called Kugelberg-Welander disease and subcategorized as IIIA and IIIB): Onset after 18 months of age. Lifespan is not affected, with wide-ranging reduction in muscle strength with a chronic course. The outcome depends primarily on the severity of muscle weakness at presentation rather than age of onset, but earlier onset tends to correlate with greater weakness. SMN2 copy number 3 or 4.
    •  Type IV (also called adult-onset SMA): Usually presents in the third decade of life and is otherwise similar to SMA type III.  SMN2 copy number 4-8.
 
The prevalence of SMA is estimated to be between 9.1 and 10 per 100,000 live births (Prior, 2010; Sugarman, 2012). In 95% cases, both copies of the SMN1 exon 7 are absent. The remaining 5% cases are compound heterozygotes for SMN1 exon 7 deletion and small intragenic variants. The molecular diagnosis of SMA consists of the detection of the absence of exons 7 of the SMN1 gene in the majority of cases (Prior, 2010).
 
Regulatory Status
 
On May 24, 2019, onasemnogene abeparvovec-xioi (e.g., Zolgensma; Avexis) was approved by the U.S. Food and Drug Administration for treatment of pediatric individuals less than 2 years of age with spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
  
The FDA has issued a black-box warning for onasemnogene abeparvovec-xioi due to the risk of acute serious liver injury and elevated aminotransferases. Individuals with pre-existing liver impairment may be at higher risk.
 
Coding
 
See CPT/HCPCS Code section below.
Policy/
Coverage:
For coverage prior to November 15, 2025, please refer policy number 2019011, Treatment for Spinal Muscular Atrophy.
 
The use of these drugs requires documentation of direct physician (MD/DO) involvement in the ordering and evaluation as well as a signature in the medical records submitted for prior approval.
 
Prior Approval will be required for Onasemnogene Abeparvovec-xioi (Zolgensma).
 
The use of this drug/therapy requires documentation of direct involvement and ordering by a physician with expertise in specified condition and in a center approved for administration of CAR-T or gene product.
 
STANDARD REVIEW FOR DURATION OF TREATMENT COURSE OR 12 MONTHS (whichever comes first). Approval timeframes may differ for members/participants of Self-Insured plans.
 
Effective June 3, 2026
 
Meets Primary Coverage Criteria Or Is Covered For Contracts Without Primary Coverage Criteria
 
Onasemnogene abeparvovec-xioi (e.g., Zolgensma) meets member benefit certificate Primary Coverage Criteria that there be scientific evidence of effectiveness in improving health outcomes or for members with contracts without Primary Coverage Criteria, is considered Medically Necessary and is covered, for the treatment of individuals with spinal muscular atrophy.
 
STANDARD REVIEW:
 
1. Individual is less than 2 years of age (Zolgensma, 2025); AND
2. individual has a  Diagnosis of spinal muscular atrophy is confirmed by genetic testing demonstrating bi-allelic mutations in the survival motor neuron 1 (SMN1) gene (Zolgensma, 2025); AND
3. Documentation of a genetic test confirms no more than  4 copies of the SMN2 gene (Zolgensma 2025); AND
4. Individuals does not have advanced spinal muscular atrophy (e.g., complete paralysis of limbs, permanent ventilator dependence) (Limitation of Use, Zolgensma 2025); AND
5. Baseline anti-adeno-associated virus serotype 9 (AAV9) antibody titers are less than or equal to 1:50 (Zolgensma, 2025); AND
6. Individual does not have ANY of the following:
a. Prior receipt of gene therapy for spinal muscular atrophy (Zolgensma, 2025); OR
b. Planned use of other survival motor neuron (SMN)-targeting therapies (for example nusinersen or risdiplam) following infusion of onasemnogene; AND
7. Onasemnogene is prescribed by a neurologist with expertise in treating spinal muscular atrophy.
 
Does Not Meet Primary Coverage Criteria Or Is Not Covered For Contracts Without Primary Coverage Criteria
 
Onasemnogene abeparvovec-xioi (e.g., Zolgensma), for any indication or circumstance not described above, does not meet member benefit certificate Primary Coverage Criteria that there be scientific evidence of effectiveness in improving health outcomes and is not covered including but not limited to:
 
1. Repeat treatment or ante-partum use of onasemnogene abeparvovec-xioi for the treatment of infantile-onset or type I spinal muscular atrophy; AND
2. Concurrent use of onasemnogene abeparvovec-xioi with nusinersen and/or risdiplam.
 
For those members without Primary Coverage Criteria, Onasemnogene abeparvovec-xioi (e.g., Zolgensma), for any indication or circumstance not described above, is considered not Medically Necessary and is not covered or is investigational. Not Medically Necessary or Investigational services are specific contract exclusions in most member benefit certificates of coverage, including but not limited to the following:
 
1. Repeat treatment or ante-partum use of onasemnogene abeparvovec-xioi for the treatment of infantile-onset or type I spinal muscular atrophy; AND
2. Concurrent use of onasemnogene abeparvovec-xioi with nusinersen and/or risdiplam.
 
Not Medically Necessary or Investigational services are specific contract exclusions in most member benefit certificates of coverage.
 
DOSAGE AND ADMINISTRATION
 
For FDA labeled indications, Onasemnogene abeparvovec-xioi (e.g., Zolgensma) must be dosed in accordance with the indication specific recommended dose per FDA label unless otherwise specified below.
 
The recommended dosage of Onasemnogene abeparvovec-xioi (e.g., Zolgensma) is 1.1 × 100 trillion vector genomes (vg) per kg of body weight. It should be administered as an intravenous infusion over 60 minutes. Systemic corticosteroids equivalent to oral prednisolone at 1 mg/kg should be administered according to the FDA approved prescribing label.
 
The FDA label states that "The safety and efficacy of Onasemnogene abeparvovec-xioi (e.g., ZOLGENSMA) in individuals with anti-AAV9 antibody titers above 1:50 have not been evaluated.” Baseline anti-AAV9 antibody testing is performed prior to infusion using. Retesting may be performed if anti-AAV9 antibody titers are reported as greater than 1:50.
 
Liver function (clinical exam, AST, ALT, total bilirubin, prothrombin time), platelet counts, and troponin-I levels should be monitored as per the prescribing label.
 
Where feasible, individual’s vaccination schedule should be adjusted to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec-xioi infusion.
 
Use of onasemnogene abeparvovec-xioi in premature neonates before reaching full term gestational age may not be recommended because concomitant treatment with corticosteroids may adversely affect neurological development.
 
Efficacy of onasemnogene abeparvovec-xioi in individuals with c.859G>C variant in SMN2 gene has not been evaluated.
 
Onasemnogene abeparvovec-xioi (e.g., Zolgensma) is available as a suspension for intravenous infusion, supplied as single-use vials.
 
Onasemnogene abeparvovec-xioi (e.g., Zolgensma) is provided in a kit containing 2 to 14 vials, as a combination of 2 vial fill volumes (either 5.5 mL or 8.3 mL). All vials have a nominal concentration of 2.0 x 10 trillion vector genomes (vg) per mL. Each vial of Onasemnogene abeparvovec-xioi (e.g., Zolgensma) contains an extractable volume of not less than either 5.5 mL or 8.3 mL.
 
Please refer to a separate policy on Site of Care or Site of Service Review (policy #2018030) for pharmacologic/biologic medications.
 
Effective November 15, 2025 to June 2, 2026
 
Meets Primary Coverage Criteria Or Is Covered For Contracts Without Primary Coverage Criteria
 
Onasemnogene abeparvovec-xioi (e.g., Zolgensma) meets member benefit certificate primary coverage criteria that there be scientific evidence of effectiveness in improving health outcomes for the treatment of individuals with infantile-onset or type I spinal muscular atrophy with a documented genetic diagnosis of spinal muscular atrophy.
 
1. Diagnosis of spinal muscular atrophy confirmed by genetic testing demonstrating bi-allelic mutations in the survival motor neuron 1 (SMN1) gene as stated below (Gene Reviews, SMA, 2020):
a. Deletion of both copies of the SMN1 gene; OR
b. Compound heterozygous mutations of the SMN1 gene (defined below):
i. Pathogenic variant(s) in both copies of the SMN1 gene; OR
ii. Pathogenic variant in one copy and deletion of the second copy of the SMN1 gene; AND
2. Documentation of onset of symptoms consistent with clinical diagnosis of type I spinal muscular atrophy less than 6 months of age (Mendell, 2017); AND
3. Documentation of a genetic test confirms no more than 2 copies of the SMN2 gene (Mendell, 2017); AND
4. Individuals is less than  6 months of age at the time of infusion of onasemnogene abeparvovec-xioi (Mendell, 2017); AND
5. Individuals does not have advanced spinal muscular atrophy (e.g., complete paralysis of limbs, permanent ventilator dependence); AND
6. Baseline anti-adeno-associated virus serotype 9 (AAV9) antibody titers less than or equal to 1:50 (FDA Zolgensma); AND
7. Prescribed by a neurologist with expertise in treating spinal muscular atrophy.
 
Note: The repeat treatment or ante-partum use of onasemnogene abeparvovec-xioi is not covered (see statement of non-coverage below).
 
Note: Concurrent use of onasemnogene abeparvovec-xioi with nusinersen and/or risdiplam is not covered (see statement of non-coverage below).
 
Does Not Meet Primary Coverage Criteria Or Is Not Covered For Contracts Without Primary Coverage Criteria
 
Onasemnogene abeparvovec-xioi (e.g., Zolgensma), for any indication or circumstance not described above, does not meet member benefit certificate primary coverage criteria that there be scientific evidence of effectiveness in improving health outcomes including but not limited to:
 
1. Repeat treatment or ante-partum use of onasemnogene abeparvovec-xioi for the treatment of infantile-onset or type I spinal muscular atrophy; AND
2. Concurrent use of onasemnogene abeparvovec-xioi with nusinersen and/or risdiplam.
 
For those members without primary coverage criteria, Onasemnogene abeparvovec-xioi (e.g., Zolgensma), for any indication or circumstance not described above, including but not limited to the following is considered investigational:
 
1. Repeat treatment or ante-partum use of onasemnogene abeparvovec-xioi for the treatment of infantile-onset or type I spinal muscular atrophy; AND
2. Concurrent use of onasemnogene abeparvovec-xioi with nusinersen and/or risdiplam.
 
Investigational services are specific contract exclusions in most member benefit certificates of coverage.
 
DOSING AND ADMINISTRATION
 
For FDA labeled indications, Onasemnogene abeparvovec-xioi (e.g., Zolgensma) must be dosed in accordance with the indication specific recommended dose per FDA label unless otherwise specified below.
 
The recommended dosage of Onasemnogene abeparvovec-xioi (e.g., Zolgensma) is 1.1 × 100 trillion vector genomes (vg) per kg of body weight. It should be administered as an intravenous infusion over 60 minutes. Systemic corticosteroids equivalent to oral prednisolone at 1 mg/kg should be administered according to the FDA approved prescribing label.
 
The FDA label states that "The safety and efficacy of Onasemnogene abeparvovec-xioi (e.g., ZOLGENSMA) in individuals with anti-AAV9 antibody titers above 1:50 have not been evaluated.” Baseline anti-AAV9 antibody testing is performed prior to infusion using. Retesting may be performed if anti-AAV9 antibody titers are reported as greater than 1:50.
 
Liver function (clinical exam, AST, ALT, total bilirubin, prothrombin time), platelet counts, and troponin-I levels should be monitored as per the prescribing label.
 
Where feasible, individual’s vaccination schedule should be adjusted to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec-xioi infusion.
 
Use of onasemnogene abeparvovec-xioi in premature neonates before reaching full term gestational age may not be recommended because concomitant treatment with corticosteroids may adversely affect neurological development.
 
Efficacy of onasemnogene abeparvovec-xioi in individuals with c.859G>C variant in SMN2 gene has not been evaluated.
 
Onasemnogene abeparvovec-xioi (e.g., Zolgensma) is available as a suspension for intravenous infusion, supplied as single-use vials.
 
Onasemnogene abeparvovec-xioi (e.g., Zolgensma) is provided in a kit containing 2 to 14 vials, as a combination of 2 vial fill volumes (either 5.5 mL or 8.3 mL). All vials have a nominal concentration of 2.0 x 10 trillion vector genomes (vg) per mL. Each vial of Onasemnogene abeparvovec-xioi (e.g., Zolgensma) contains an extractable volume of not less than either 5.5 mL or 8.3 mL.
 
Please refer to a separate policy on Site of Care or Site of Service Review policy #2018030 for pharmacologic/biologic medications. It will be expected that this therapy will be administered through a certified treatment location. Please see: http://www.curesma.org/zolgensma-administration-sites.html for locations of administration.
Rationale:
Type 0 or IV Spinal Muscular Atrophy
There are currently no studies assessing the efficacy and safety of nusinersen in patients with type 0 or IV spinal muscular atrophy.
 
Onasemnogene Abeparvovec-Xioi
 
The clinical development program of onasemnogene abeparvovec-xioi for patients with symptomatic spinal muscular atrophy type I includes 4 prospective cohort studies; 2 dose-finding study, 2 phase 3 confirmatory studies (STRIVE-US: Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1; STRIVE EU: Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 ), and 1 long-term follow-up study (START: Long-Term Follow-up Study for Patients From AVXS-101-CL-101). These trials will enroll a total of 65 patients with symptomatic spinal muscular atrophy type I. Of these 4 trials, only the dose-finding phase 1 study has been completed and has reported 2-year follow-up data. (Mendell JR, et al. 2017);(Al-Zaidy S, Kolb SJ, et al. 2019); (Al-Zaidy S, Pickard AS, et al. 2019); Lowes LP, et al. 2019)
 
In the phase 1 study, 12 of 15 infants received the proposed therapeutic dose while 3 received a minimally effective dose. At a median follow-up ranging from 30.7 to 27.8 months (based on 2 dose cohorts), all 15 patients survived and none of the 12 patients who received the proposed therapeutic dose required permanent ventilation at the 2-year follow-up. Based on the known natural history of patients with spinal muscular atrophy type I with 2 copies of the SMN2 gene, 8% of patients are expected to survive beyond 2 years without ventilation. In terms of motor functions, all 12 patients achieved at least 1 motor milestone, with 92% of those achieving scores greater than 40 on the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND). Compared with the known natural history, attaining a CHOP INTEND score greater than 40 is rare (Finkel RS, et al. 2016), and patients experience a 10.7-point drop in CHOP INTEND scores between 6 to 12 months of age. Treatment-related asymptomatic transient elevated liver function enzymes (categorized as serious adverse events) were reported in 2 patients. The early data in a small number of patients diagnosed with spinal muscular atrophy type I is indicative of positive impact on survival and motor functions that are durable up to 2 years.
 
The FDA approval was based on a pooled analysis of 21 patients with 2 copies of SMN2 from the pivotal phase I and STRIVE-US trial with a data analysis cut off of March 2019. Efficacy was established on the basis of survival, and achievement of developmental motor milestones such as sitting without support. Comparison of the results of the ongoing clinical trial to available natural history data of patients with infantile-onset spinal muscular atrophy was the primary evidence for effectiveness of onasemnogene abeparvovec-xioi.
 
While the current evidence for symptomatic type I spinal muscular atrophy patients is limited to patients with 2 copies of SMN2, approximately 20% of type I spinal muscular atrophy patients may have 3 copies of SMN2 (Feldkotter M, et al. 2002). Given the treatment effect observed in symptomatic patients, it is possible that patients with 3 copies of SMN2 may experience a clinically meaningful benefit. However, there is no published evidence to support such a hypothesis. Further, there is no published data that supports clinical benefit in Type I spinal muscular atrophy patients who are administered onasemnogene abeparvovec-xioi after 6 months of age.
 
ICER Report
In summarizing the uncertainties of the clinical evidence, the ICER Report noted considerable uncertainty in the generalizability of the results and in the long-term durability and tolerability of treatment. Further, the Report notes additional uncertainty related to the possibility of loss of transgene expression over time and subsequent treatment pathway. The Report also noted that some patients in the pivotal trial subsequently received nusinersen, but the effects of combination or sequential therapies have not been well studied.
 
2026 Update
The postmarketing study NCT04851873 has evaluated safety of onasemnogene was evaluated in 24 children, aged between 1.5 to 9.1 years (median age, 4.9 years), with weight range from ≥ 8.5 kg to ≤ 21 kg (median weight, 15.8 kg). Only one of the 24 patients was under the age of 2 years at the time of onasemnogene administration. Patients in Study 3 had 2 to 4 copies of SMN2. Before treatment with onasemnogene, 21 patients discontinued their previous treatment with nusinersen or risdiplam. The types of adverse reactions observed in Study 3 were consistent with those of Studies 1 and 2. Liver enzyme increases in Study 3 occurred at a higher frequency compared with the previous 4 studies. AST or ALT elevations > 2 × upper limit of normal were observed in the majority of patients (23 out of 24 patients), including 21 patients with ALT elevations > 3 × upper limit of normal and 5 patients with ALT elevations > 20 × upper limit of normal. These patients were clinically asymptomatic and there were no elevations of bilirubin. The AST and ALT elevations were managed with the use of corticosteroids, typically with prolonged duration and/or given at a higher dose. Transient decreases in platelet counts, which met the criteria for thrombocytopenia were observed in 20 out of 24 patients. Four patients had platelet counts below 50,000 per µL. (McMillan, 2024)
CPT/HCPCS:
C9399Unclassified drugs or biologicals
J3399Injection, onasemnogene abeparvovec xioi, per treatment, up to 5x10 15 vector genomes
J3490Unclassified drugs
J3590Unclassified biologics
References:
AI-Zaidy S, Pickard AS, Katha K, et al.(2019) Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. Feb 2019; 54(2): 179-185. PMID 30548438

AI-Zaidy SA, Kolb SJ, Lowes L, et al.(2019) AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019; 6(3): 307-317. PMID 31381526

Bodamer, OA,(2021) Spinal Muscular Atrophy, UpToDate August, 2021

Feldkotter M, Schwarzer V, Wirth R, et al.(2002) Quantitative analyses of SMN1 and SMN2 based on real-time light Cycler PCR: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy. Am J Hum Genet. Feb 2002;70(2):358-368. PMID 11791208

Finkel RS, McDermott MP, Kaufmann P, et al.(2014) Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology. Aug 26 2014;83(9):810-817. PMID 25080519

Foust KD, Nurre E, Montgomery CL, et al.(2009) lntravascular MV 9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol. Jan 2009; 27(1 ): 59-65. PMID 19098898

Glanzman AM, Mazzone E, Main M, et al.(2010) The Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): test development and reliability. Neuromuscul Disord. Mar 2010;20(3):155-161. PMID 20074952

Institute for Clinical and Evidence Review.(2019) Spinraza and Zolgensma for Spinal Muscular Atrophy: Effectiveness and Value Final Evidence Report April 3, 2019; Updated May 24, 2019. https://icer-review.org/wp-content/uploads/2018/07/ICER_SMA_Final_Evidence_Report_052419.pdf. Accessed May 28, 2019.

Kolb SJ, Kissel JT.(2015) Spinal Muscular Atrophy. Neuol Clin 2015 Nov; 33(4):831-46: 10.106/j.ncl.2015.07.004

Lefebvre S, Burlet P, Liu Q, et al.(1997) Correlation between severity and SMN protein level in spinal muscular atrophy. Correlation between severity and SMN protein level in spinal muscular atrophy.

Lorson CL, Hahnen E, Androphy EJ, et al.(1999) A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy. Proc Natl Acad Sci U S A. May 25 1999;96(11):6307-6311. PMID 10339583

Lowes LP, Alfano LN, Arnold WD, et al.(2019) Impact of Age and Motor Function in a Phase 1/2A Study of Infants with SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neural. Sep 2019; 98: 39-45. PMID 31277975

Mendell JR, Al-Zaidy S, Shell R, et al.(2017) Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy N Engl J Med 2017; 377(18):1713-22.

Mercuri E, Darras BT, Chiriboga CA, et al.(2018) Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy. N Engl J Med. Feb 15 2018; 378(7): 625-635. PMID 29443664

National Institute for Health and Care Excellence (NICE)(2019) Technology appraisal guidance: Nusinersen for treating spinal muscular atrophy. Technology appraisal guidance: Nusinersen for treating spinal muscular atrophy.

Package Insert(2019) Zolgensma (onasemnogene abeparvovec-xioi) Bannockburn, IL: AveXis, Inc.; 2019.

Package Insert(2025) Zolgensma Bannockburn, IL: AveXis, Inc.; 2025.

Prior TW, Krainer AR, Hua Y, et al.(2009) A positive modifier of spinal muscular atrophy in the SMN2 gene. Am J Hum Genet. Sep 2009; 85(3): 408-13. PMID 19716110

Prior TW, Snyder PJ, Rink BD, et al.(2010) Newborn and carrier screening for spinal muscular atrophy. Am J Med Genet A. Jul 2010;152A(7):1608-1616. PMID 20578137

Spinal Muscular Atrophy(2017) National Organization for Rare Disorders, Russman B https://rarediseases.org/rare-diseases/spinal-muscular-atrophy/. Accessed January 18, 2017.

Spinal Muscular Atrophy(2020) Muscular Dystrophy Association https://www.mda.org/disease/spinal-muscular­atrophy. Accessed March 12, 2020

Sugarman EA, Nagan N, Zhu H, et al.(2012) Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72,400 specimens. Eur J Hum Genet. Jan 2012;20(1):27-32. PMID 21811307

Thomas W Prior, PhD, FACMG, Meganne E Leach, MSN, PNP, and Erika Finanger, MD,(2020) Gene Reviews: Spinal Muscular Atrophy, Initial Posting: February 24, 2000; Last Revision: December 3, 2020. Accessed: September 29, 2021.

Valeri CF, Ning K, Wyles M, et al.(2010) Systemic delivery of scMV 9 expressing SMN prolongs survival in a model of spinal muscular atrophy Sci Transl Med. Jun 09 2010; 2(35): 35ra42. PMID 20538619

Wang CH, Finkel RS, Bertini ES, et al.(2007) Consensus statement for standard of care in spinal muscular atrophy. J Child Neurol. Aug 2007;22(8):1027-1049. PMID 17761659

Website(2019) Cure SMA Available at: http://www.curesma.org/sma/. Accessed June 17, 2019

Zerres K, Rudnik-Schoneborn S(1995) Natural history in proximal spinal muscular atrophy. Clinical analysis of 445 patients and suggestions for a modification of existing classifications. Arch Neurol. May 1995;52(5):518-523.
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