Plasmodium vivax epidemiology in Ethiopia 2000-2020: A systematic review and meta-analysis

Background Ethiopia is one of the scarce rare African countries where Plasmodium vivax and P. falciparum co-exist. There has been no attempt to derive a robust prevalence estimate of P. vivax in the country although a clear understanding of the epidemiology of this parasite is essential for informed decisions. This systematic review and meta-analysis, therefore, is aimed to synthesize the available evidences on the distribution of P. vivax infection by different locations/regions, study years, eco-epidemiological zones, and study settings in Ethiopia. Methods This study was conducted in accordance with Preferred Reposting Items for Systematic Reviews and Meta Analyses (PRISMA) guidelines. Studies conducted and published over the last two decades (2000 to 2020) that reported an estimate of P. vivax prevalence in Ethiopia were included. The Cochrane Q (χ2) and the I2 tests were used to assess heterogeneity, and the funnel plot and Egger’s test were used to examine publication bias. A p-value of the χ2 test <0.05 and an I2 value >75% were considered presence of considerable heterogeneity. Random effect models were used to obtain pooled estimate of P. vivax infection prevalence. This study is registered with PROSPERO (International Prospective Register of Systematic Reviews): ID CRD42020201761. Results We screened 4,932 records and included 79 studies that enrolled 1,676,659 confirmed malaria cases, from which 548,214 (32.69%) were P. vivax infections and 1,116,581 (66.59%) were due to P. falciparum. The rest (11,864 or 0.7%) were due to mixed infections. The pooled estimate of P. vivax prevalence rate was 8.93% (95% CI: 7.98–9.88%) with significant heterogeneity (I2 = 100%, p<0.0001). Regional differences showed significant effects (p<0.0001, and I2 = 99.4%) on the pooled prevalence of P. vivax, while study years (before and after the scaling up of interventional activities) did not show significant differences (p = 0.9, I2 = 0%). Eco-epidemiological zones considered in the analysis did show a significant statistical effect (p<0.001, I2 = 78.5%) on the overall pooled estimate prevalence. Also, the study setting showed significant differences (p = 0.001, and I2 = 90.3%) on the overall prevalence, where significant reduction of P. vivax prevalence (4.67%, 95%CI: 1.41–7.93%, p<0.0001) was observed in studies conducted at the community level. The studies included in the review demonstrated lack of publication bias qualitatively (symmetrical funnel plot) and quantitatively [Egger’s test (coefficient) = -2.97, 95% CI: -15.06–9.13, p = 0.62]. Conclusion The estimated prevalence of P. vivax malaria in Ethiopia was 8.93% with P. vivax prevailing in the central west region of Ethiopia, but steadily extending to the western part of the country. Its distribution across the nation varies according to geographical location, study setting and study years.

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Prevalence of pre-stroke depression and its association with post-stroke depression: a systematic review and meta-analysis

Psychological Medicine ◽

10.1017/s0033291718002003 ◽

2018 ◽

Vol 49 (4) ◽

pp. 685-696 ◽

Cited By ~ 7

Author(s):

Martin Taylor-Rowan ◽

Oyiza Momoh ◽

Luis Ayerbe ◽

Jonathan J. Evans ◽

David J. Stott ◽

...

Keyword(s):

Systematic Review ◽

Publication Bias ◽

Meta Analysis ◽

Assessment Method ◽

Self Report ◽

Funnel Plot ◽

Strength Of Evidence ◽

Post Stroke ◽

Depression Assessment ◽

Post Stroke Depression

AbstractBackgroundDepression is a common post-stroke complication. Pre-stroke depression may be an important contributor, however the epidemiology of pre-stroke depression is poorly understood. Using systematic review and meta-analysis, we described the prevalence of pre-stroke depression and its association with post-stroke depression.MethodsWe searched multiple cross-disciplinary databases from inception to July 2017 and extracted data on the prevalence of pre-stroke depression and its association with post-stroke depression. We assessed the risk of bias (RoB) using validated tools. We described summary estimates of prevalence and summary odds ratio (OR) for association with post-stroke depression, using random-effects models. We performed subgroup analysis describing the effect of depression assessment method. We used a funnel plot to describe potential publication bias. The strength of evidence presented in this review was summarised via ‘GRADE’.ResultsOf 11 884 studies identified, 29 were included (total participantsn= 164 993). Pre-stroke depression pooled prevalence was 11.6% [95% confidence interval (CI) 9.2–14.7]; range: 0.4–24% (I295.8). Prevalence of pre-stroke depression varied by assessment method (p= 0.02) with clinical interview suggesting greater pre-stroke depression prevalence (~17%) than case-note review (9%) or self-report (11%). Pre-stroke depression was associated with increased odds of post-stroke depression; summary OR 3.0 (95% CI 2.3–4.0). All studies were judged to be at RoB: 59% of included studies had an uncertain RoB in stroke assessment; 83% had high or uncertain RoB for pre-stroke depression assessment. Funnel plot indicated no risk of publication bias. The strength of evidence based on GRADE was ‘very low’.ConclusionsOne in six stroke patients have had pre-stroke depression. Reported rates may be routinely underestimated due to limitations around assessment. Pre-stroke depression significantly increases odds of post-stroke depression.Protocol identifierPROSPERO identifier: CRD42017065544

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The association between lithium in drinking water and neuropsychiatric outcomes: A systematic review and meta-analysis from across 2678 regions containing 113 million

Australian & New Zealand Journal of Psychiatry ◽

10.1177/0004867420963740 ◽

2020 ◽

pp. 000486742096374 ◽

Cited By ~ 1

Author(s):

Brenton Eyre-Watt ◽

Eesharnan Mahendran ◽

Shuichi Suetani ◽

Joseph Firth ◽

Steve Kisely ◽

...

Keyword(s):

Systematic Review ◽

Drinking Water ◽

Confidence Interval ◽

Publication Bias ◽

Hospital Admissions ◽

Meta Analysis ◽

Significant Heterogeneity ◽

Suicide Rates ◽

Registration Number ◽

Trim And Fill

Background: Lithium in drinking water may have significant mental health benefits. We investigated the evidence on the association between lithium concentrations in drinking water and their neuropsychiatric outcomes. Methods: We conducted a systematic review and meta-analysis and searched Pubmed, Embase, Web of Science, PsycINFO and CINAHL up to 19 January 2020, for peer-reviewed research examining the association between lithium concentrations in drinking water and neuropsychiatric outcomes. We used a pairwise analysis and a random effects model to meta-analyse suicide rates and psychiatric hospital admissions. We assessed for publication bias using Egger’s test and Duval and Tweedie’s Trim and Fill analysis. Results: Twenty-seven studies including 113 million subjects were included in this systematic review. Meta-analysis of 14 studies including 94 million people found higher lithium concentrations were associated with reduced suicide rates ( r = −0.191, 95% confidence interval = [−0.287, −0.090], p < 0.001) and meta-analysis of two studies including 5 million people found higher lithium concentrations were associated with fewer hospital admissions ( r = −0.413, 95% confidence interval = [−0.689, −0.031], p = 0.035). We found significant heterogeneity between studies ( Q = 67.4, p < 0.001, I2 = 80.7%) and the presence of publication bias (Egger’s test; t value = 2.90, p = 0.013). Other included studies did not provide sufficient data to analyse other neuropsychiatric outcomes quantitatively. Conclusion: Higher lithium concentrations in drinking water may be associated with reduced suicide rates and inpatient psychiatric admissions. The relationship with other neuropsychiatric outcomes and complications remains unclear. Further research is required before any public health recommendations can be made. Trial registration number: The study was registered with PROSPERO, number CRD42018090145.

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The impact of time to adjuvant chemotherapy (AC) on survival in colorectal cancer (CRC): A systematic review and meta-analysis.

Journal of Clinical Oncology ◽

10.1200/jco.2011.29.4_suppl.364 ◽

2011 ◽

Vol 29 (4_suppl) ◽

pp. 364-364 ◽

Cited By ~ 4

Author(s):

J. J. Biagi ◽

M. Raphael ◽

W. D. King ◽

W. Kong ◽

W. J. Mackillop ◽

...

Keyword(s):

Systematic Review ◽

Prognostic Factors ◽

Adjuvant Chemotherapy ◽

Publication Bias ◽

Meta Analysis ◽

Disease Free Survival ◽

Significant Heterogeneity ◽

Risk Of Death ◽

The Impact ◽

The Relationship

364 Background: The optimal timing from CRC surgery to initiation of AC is unknown. We report a systematic review and meta-analysis to determine the relationship between time to adjuvant chemotherapy (TTAC) and survival. Methods: A systematic review of literature was done to identify studies that described the relationship between TTAC and survival. Studies were only included if the distribution of relevant prognostic factors was adequately described, and either comparative groups were balanced or results adjusted for the prognostic factors. Hazard ratio (HR) and TTAC for overall survival (OS) and disease free survival (DFS) from each study were converted to a regression coefficient (β) and standard error (SE) corresponding to a continuous representation per 4 weeks of TTAC. The adjusted β from individual studies were combined using a fixed-effect model. Inverse-variance (1/SE2) was used to weight individual studies. The possible effect of publication bias was investigated using the trim and fill approach. Results: We identified 9 eligible studies involving 14,357 patients (4 published articles, 5 abstracts). Two studies were randomized trials and 7 were cohort studies. Six studies reported TTAC as a binary variable and 3 reported TTAC as ≥3 categories. An estimate of HR for OS was derived from all 9 studies and estimate for DFS was derived from 5 studies. Meta-analysis demonstrated that a 4-week increase in TTAC was associated with a significant decrease in both OS (HR = 1.12, 95% CI 1.09-1.15), and DFS (HR = 1.15, 95% CI 1.11-1.20). The analysis showed no significant heterogeneity among studies. These TTAC associations remained significant after analysis for potential publication bias, and when the analysis was repeated excluding the two studies of largest weight. Conclusions: This study demonstrates a 12% increase in the risk of death for each 4 week of delay in the start of AC for CRC. These findings indicate the need for clinicians and health systems managers to take the steps necessary to keep TTAC as short as reasonably achievable. In addition, our results suggest there may be some benefit to AC after a 3-month TTAC delay. No significant financial relationships to disclose.

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Depression and its Main Determinants Among Iranian Operating Room Personnel: A Systematic Review and Meta-Analysis

International Clinical Neuroscience Journal ◽

10.15171/icnj.2018.16 ◽

2018 ◽

Vol 5 (3) ◽

pp. 81-85

Author(s):

Mehdi Ameri ◽

Mohammad Reza Hosseini Nodoushan ◽

Amir Shahbazzadeh ◽

Mehran Arab Ahmadi

Keyword(s):

Systematic Review ◽

Operating Room ◽

Systematic Reviews ◽

Meta Analysis ◽

Severe Depression ◽

Significant Heterogeneity ◽

Operating Room Personnel ◽

Severe Condition ◽

Prevalence Of Depression ◽

Main Determinants

Background: Most nurses, especially operating room personnel, seems to be more likely to be affected by mood disorders than other social strata. The present study attempted to systematically review the prevalence of depression and its main determinants among operating room personnel in Iran. Methods: The method of this systematic review is documenting in a published protocol in the International Prospective Register of Systematic Reviews (PROSPERO) and the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) checklist. After this massive search, titles and abstracts of retrieved documents have screened and all irrelevant articles excluded. Two reviewers screened the documents and selected all relevant studies and assessed included articles separately. Results: Totally, 12 citations found in the initial literature search where four citations excluded, as they did not meet the inclusion criteria. The final number of studies available for analysis was 12 including a total of 373 operating room personnel (86 men and 287 women, mean the age of 27.71 years ranged from 20 to 36 years). The pooled prevalence of depression among operating room personnel was estimated to be 45.3%. In this regard, 27.0% of personnel suffered from severe depression. A significant heterogeneity found in the overall analysis of the overall prevalence of depression and its severe pattern. Conclusion: A notable number of operating room personnel in Iran suffer from depression even in its severe condition emphasizing the importance of the managerial approach to minimize its adverse effects on their performance as well as to improve their quality of life

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Vancomycin-Resistant Enterococci (VRE) in Nigeria: The First Systematic Review and Meta-Analysis

Antibiotics ◽

10.3390/antibiotics9090565 ◽

2020 ◽

Vol 9 (9) ◽

pp. 565

Author(s):

Yusuf Wada ◽

Azian Binti Harun ◽

Chan Yean Yean ◽

Abdul Rahman Zaidah

Keyword(s):

Systematic Review ◽

Publication Bias ◽

Meta Analysis ◽

Forest Plot ◽

Funnel Plot ◽

Vancomycin Resistant Enterococci ◽

Pooled Prevalence ◽

Vancomycin Resistant ◽

Meta Regression ◽

Leave One Out

Vancomycin-Resistant Enterococci (VRE) are on the rise worldwide. Here, we report the first prevalence of VRE in Nigeria using systematic review and meta-analysis. International databases MedLib, PubMed, International Scientific Indexing (ISI), Web of Science, Scopus, Google Scholar, and African journals online (AJOL) were searched. Information was extracted by two independent reviewers, and results were reviewed by the third. Two reviewers independently assessed the study quality using the Preferred Reporting Items for Systematic Reviews and Meta-Analysis (PRISMA) checklist. OpenMeta analyst was used. The random effect was used, and publication bias was assessed using a funnel plot. Between-study heterogeneity was assessed, and the sources were analysed using the leave-one-out meta-analysis, subgroup analysis, and meta-regression. Nineteen studies met the eligibility criteria and were added to the final meta-analysis, and the study period was from 2009–2018. Of the 2552 isolates tested, 349 were VRE, and E. faecalis was reported the most. The pooled prevalence of VRE in Nigeria was estimated at 25.3% (95% CI; 19.8–30.8%; I2 = 96.26%; p < 0.001). Between-study variability was high (t2 = 0.011; heterogeneity I2 = 96.26% with heterogeneity chi-square (Q) = 480.667, degrees of freedom (df) = 18, and p = 0.001). The funnel plot showed no publication bias, and the leave-one-out forest plot did not affect the pooled prevalence. The South-East region had a moderate heterogeneity though not significant (I2 = 51.15%, p = 0.129). Meta-regression showed that all the variables listed contributed to the heterogeneity except for the animal isolate source (p = 0.188) and studies that were done in 2013 (p = 0.219). Adherence to proper and accurate antimicrobial usage, comprehensive testing, and continuous surveillance of VRE are required.

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Prevalence and determinants of ectopic pregnancy in Ethiopia: Systematic review and meta-analysis

Journal of Endometriosis and Pelvic Pain Disorders ◽

10.1177/22840265211062010 ◽

2022 ◽

pp. 228402652110620

Author(s):

Asteray Ayenew

Keyword(s):

Systematic Review ◽

Ectopic Pregnancy ◽

Publication Bias ◽

Meta Analysis ◽

Case Control Studies ◽

Major Health Problem ◽

Funnel Plot ◽

Sexually Transmitted ◽

Determinant Factors ◽

History Of

Background: Ectopic pregnancy is a life-threatening obstetric emergency, and is a major health problem for women of fertile age. Therefore, the aim of this systematic review and meta-analysis was to estimate the prevalence, determinant factors, and outcomes of ectopic pregnancy among fertile age women in Ethiopia. Method: International databases (MEDLINE/Pub Med, Hinari, Scopus, Google scholar, African journals, and literatures were searched and nine eligible cross sectional and two case control studies were included in this systematic review and meta-analysis. Eggers test and funnel plot were computed to check publication bias across the studies. Publication bias was computed using a funnel plot and eggers test. Heterogeneity of the studies was checked using Cochrane Q-test and I2 statistic. Results: The pooled prevalence of ectopic pregnancy in Ethiopia was 3.61% (95%CI: 2.24–4.98, I2 = 89.2.0%, p < 0.001). Having cesarean section scar (AOR = 7.44, 95%CI: 5.48–10.09), single marital status (AOR = 5.71, 95%CI: 4.76–6.85), history of sexually transmitted infection (AOR = 4.68, 95%CI: 3.04–7.19), history of abortion (AOR = 3.89, 95%CI: 3.35–4.52), history of ectopic pregnancy (AOR = 5.74, 95%CI: 3.81–8.65), and emergency contraceptive use (AOR = 8.72, 95%CI: 2.90–26.20) were the determinant factors for the occurrence of ectopic pregnancy. Conclusion: This systematic review and meta-analysis showed that the prevalence of ectopic pregnancy was high in Ethiopia. Thus, educating women to limit the number of sexual partners, smoking cessation, using a condom during sex helps prevent sexually transmitted infections and the risk of pelvic inflammatory disease is crucial.

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Methodology and reporting of systematic reviews and meta-analyses of observational studies in psychiatric epidemiology: Systematic review

The British Journal of Psychiatry ◽

10.1192/bjp.bp.111.098103 ◽

2012 ◽

Vol 200 (6) ◽

pp. 446-453 ◽

Cited By ~ 23

Author(s):

Traolach S. Brugha ◽

Ruth Matthews ◽

Zoe Morgan ◽

Trevor Hill ◽

Jordi Alonso ◽

...

Keyword(s):

Systematic Review ◽

Systematic Reviews ◽

Meta Analysis ◽

Epidemiological Studies ◽

Vital Role ◽

Primary Study ◽

Synthesis Methods ◽

Significant Heterogeneity ◽

Many Sources ◽

Meta Analyses

BackgroundRelatively little is known of the use of systematic review and synthesis methods of non-randomised psychiatric epidemiological studies, which play a vital role in aetiological research, planning and policy-making.AimsTo evaluate reviews of psychiatric epidemiological studies of functional mental disorders that employed synthesis methods such as systematic review or meta-analysis, or other forms of quantitative review.MethodWe searched the literature to identify appropriate reviews published during the period 1996 to April 2009. Selected reviews were evaluated using published review guidelines.ResultsWe found 106 reviews in total, of which 38 (36%) did not mention method of data abstraction from primary studies at all. Many failed to mention study quality, publication bias, bias and confounding. In 73 studies that performed a meta-analysis, 58 (79%) tested for heterogeneity and of these, 47 found significant heterogeneity. Studies that detected heterogeneity made some allowance for this. A major obstacle facing reviewers is the wide variation between primary studies in the use of instruments to measure outcomes and in sampling methods used.ConclusionsMany deficiencies found in systematic reviews are potentially remediable, although synthesis of primary study findings in a field characterised by so many sources of heterogeneity will remain challenging.

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Effects of high-fidelity simulation based on life-threatening clinical condition scenarios on learning outcomes of undergraduate and postgraduate nursing students: a systematic review and meta-analysis

BMJ Open ◽

10.1136/bmjopen-2018-025306 ◽

2019 ◽

Vol 9 (2) ◽

pp. e025306 ◽

Cited By ~ 14

Author(s):

Carmen La Cerra ◽

Angelo Dante ◽

Valeria Caponnetto ◽

Ilaria Franconi ◽

Elona Gaxhja ◽

...

Keyword(s):

Systematic Review ◽

Nursing Students ◽

Learning Outcomes ◽

Systematic Reviews ◽

Clinical Condition ◽

Meta Analysis ◽

High Fidelity ◽

Significant Heterogeneity ◽

Life Threatening ◽

And Performance

ObjectiveThe purpose was to analyse the effectiveness of high-fidelity patient simulation (HFPS) based on life-threatening clinical condition scenarios on undergraduate and postgraduate nursing students’ learning outcomes.DesignA systematic review and meta-analysis were conducted based on the Cochrane Handbook for Systematic Reviews of Interventions and its reporting was checked against the Preferred Reporting Items for Systematic Reviews and Meta-Analyses checklist.Data sourcesPubMed, Scopus, CINAHL with Full Text, Wiley Online Library and Web of Science were searched until July 2017. Author contact, reference and citation lists were checked to obtain additional references.Study selectionTo be included, available full-texts had to be published in English, French, Spanish or Italian and (a) involved undergraduate or postgraduate nursing students performing HFPS based on life-threatening clinical condition scenarios, (b) contained control groups not tested on the HFPS before the intervention, (c) contained data measuring learning outcomes such as performance, knowledge, self-confidence, self-efficacy or satisfaction measured just after the simulation session and (d) reported data for meta-analytic synthesis.Review methodThree independent raters screened the retrieved studies using a coding protocol to extract data in accordance with inclusion criteria.Synthesis methodFor each study, outcome data were synthesised using meta-analytic procedures based on random-effect model and computing effect sizes by Cohen’s d with a 95% CI.ResultsThirty-three studies were included. HFPS sessions showed significantly larger effects sizes for knowledge (d=0.49, 95% CI [0.17 to 0.81]) and performance (d=0.50, 95% CI [0.19 to 0.81]) when compared with any other teaching method. Significant heterogeneity among studies was detected.ConclusionsCompared with other teaching methods, HFPS revealed higher effects sizes on nursing students’ knowledge and performance. Further studies are required to explore its effectiveness in improving nursing students’ competence and patient outcomes.

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Prevalence of HIV and Syphilis and their Co-infection among Men Having Sex with Men in Asia: A Systematic Review and Meta-analysis

10.1101/2021.12.21.21268191 ◽

2021 ◽

Author(s):

Sultan Mahmud ◽

Md Mohsin ◽

Abdul Muyeed ◽

Sorif Hossain ◽

Md Mynul Islam ◽

...

Keyword(s):

Publication Bias ◽

Meta Analysis ◽

Random Effect ◽

Random Effect Model ◽

Final Analysis ◽

Asia Pacific ◽

Infection Prevalence ◽

Significant Heterogeneity ◽

Sex With Men ◽

Very High

Background: Studies found that the group of men who have sex with men (MSM) is at a very high level of risk of HIV and sexually transmitted infections (STIs) in Asian regions due to multiple reasons. Although the prevalence of HIV among general people in Asia is considered low, the prevalence of HIV and Syphilis among MSM in this region was found very high and usually, it goes unnoticed. This study aimed to inspect the prevalence and trends of HIV, Syphilis, and their co-infection among MSM in Asia. Methods: A systematic search was performed on January 5, 2021, in PubMed, MEDLINE, and Google Scholar databases. To evaluate the heterogeneity, Q-tests and were used. To explore the publication bias, Eggers' test and funnel plot were used. The random-effect model and subgroup analysis were performed due to the significant heterogeneity. Results: A total of 2,872 articles were identified, and 66 articles of high quality were included in the final analysis. The overall prevalence of HIV among MSM and Syphilis was estimated considering 69 estimates from 66 studies whereas 19 estimates of co-infection were found in 17 studies. The pooled HIV prevalence was 8.48% (CI: 7.01-9.95) and the pooled Syphilis prevalence was 9.86% (CI: 8.30-11.41) with significant heterogeneity and publication bias. The pooled prevalence of HIV and Syphilis co-infection was 2.99% (CI: 1.70-4.27) with significant heterogeneity and no publication bias. The HIV, Syphilis, and HIV and Syphilis co-infection prevalence estimates exhibited an upward trend during 2002-2017. Conclusions: HIV, Syphilis, and their co-infection are quite prevalent among MSM in the Asia-Pacific region. Integrated and intensified intervention strategies, HIV testing, and improved access to antiretroviral treatment as well as increased awareness are needed to reduce HIV, Syphilis, and their co-infection among the discussed vulnerable group.

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Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models

Gene Therapy ◽

10.1038/s41434-021-00292-4 ◽

2021 ◽

Author(s):

Ellie M. Chilcott ◽

Evalyne W. Muiruri ◽

Theodore C. Hirst ◽

Rafael J. Yáñez-Muñoz

Keyword(s):

Systematic Review ◽

Experimental Design ◽

Spinal Muscular Atrophy ◽

Publication Bias ◽

Median Survival ◽

Muscular Atrophy ◽

Meta Analysis ◽

Significant Heterogeneity ◽

Rodent Models ◽

Genetic Therapy

AbstractSpinal muscular atrophy (SMA) is a severe childhood neuromuscular disease for which two genetic therapies, Nusinersen (Spinraza, an antisense oligonucleotide), and AVXS-101 (Zolgensma, an adeno-associated viral vector of serotype 9 AAV9), have recently been approved. We investigated the pre-clinical development of SMA genetic therapies in rodent models and whether this can predict clinical efficacy. We have performed a systematic review of relevant publications and extracted median survival and details of experimental design. A random effects meta-analysis was used to estimate and compare efficacy. We stratified by experimental design (type of genetic therapy, mouse model, route and time of administration) and sought any evidence of publication bias. 51 publications were identified containing 155 individual comparisons, comprising 2573 animals in total. Genetic therapies prolonged survival in SMA mouse models by 3.23-fold (95% CI 2.75–3.79) compared to controls. Study design characteristics accounted for significant heterogeneity between studies and greatly affected observed median survival ratios. Some evidence of publication bias was found. These data are consistent with the extended average lifespan of Spinraza- and Zolgensma-treated children in the clinic. Together, these results support that SMA has been particularly amenable to genetic therapy approaches and highlight SMA as a trailblazer for therapeutic development.

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