Evidence map›Paper›PMID 21482919›Full record

ArticleArchives of neurology2011

Spinal muscular atrophy: a timely review.

Stephen J Kolb, John T Kissel

Registry-linked trialAbstract readHistorical Article
In one paragraph

Article in Archives of neurology, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01736553 (Spinal Muscular Atrophy), which is not on this map. Cited by 155 papers, 5 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
155citing papers in PubMed, 5 pooled it
8.2field-weighted citation impact, top 2% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT01736553 completednot on this mapstarted 2012, after this paper: background citation

Spinal Muscular Atrophy (SMA) Biomarkers in the Immediate Postnatal Period of Development

TypeobservationalSponsorOhio State UniversityRan2012 to 2015Enrolled53ConditionsSpinal Muscular Atrophy (SMA)
3 · Its place in the literature

Who cites it

155 citing papers in PubMed, 5 syntheses or guidelines pooled it, 328 citations in OpenAlex.

  1. Proteomic alterations in cerebrospinal fluid of spinal muscular atrophy patients undergoing nusinersen therapy: a systematic review and meta-analysis of potential biomarkers of treatment response.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2026
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  18. Wearable Bioelectronics for Home-Based Monitoring and Treatment of Muscle Atrophy.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025
    Review
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95 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

2 authors at 1 institution in 1 country.

Stephen J KolbDepartment of Neurology, Ohio State University, Columbus, 43210, USA.
John T Kissel
The Ohio State University · US

Funding

University of Iowa Clinical and Translational Science Program (TL1)UL1RR025755 · NCRR · OHIO STATE UNIVERSITY · PI JACKSON, REBECCA D · 2008 to 2011
$26.3M
Functional consequences of HSPB1 mutations that result in motor neuron diseaseK08NS067282 · NINDS · OHIO STATE UNIVERSITY · PI KOLB, STEPHEN J. · 2010 to 2014
$881k
NCRR NIH HHS UL1 RR025755NINDS NIH HHS K08 NS067282
6 · The paper itself

Abstract

Spinal muscular atrophy (SMA) is a neurodegenerative disease characterized by loss of motor neurons in the anterior horn of the spinal cord and resultant weakness. The most common form of SMA, accounting for 95% of cases, is autosomal recessive proximal SMA associated with mutations in the survival of motor neurons (SMN1) gene. Relentless progress during the past 15 years in the understanding of the molecular genetics and pathophysiology of SMA has resulted in a unique opportunity for rational, effective therapeutic trials. The goal of SMA therapy is to increase the expression levels of the SMN protein in the correct cells at the right time. With this target in sight, investigators can now effectively screen potential therapies in vitro, test them in accurate, reliable animal models, move promising agents forward to clinical trials, and accurately diagnose patients at an early or presymptomatic stage of disease. A major challenge for the SMA community will be to prioritize and develop the most promising therapies in an efficient, timely, and safe manner with the guidance of the appropriate regulatory agencies. This review will take a historical perspective to highlight important milestones on the road to developing effective therapies for SMA.

Indexed as

AnimalsDisease Models, AnimalGenetic Predisposition to DiseaseGenetic TherapyHistory, 19th CenturyHistory, 20th CenturyHistory, 21st CenturyHumansMuscular Atrophy, SpinalMutationSurvival of Motor Neuron 1 ProteinSMN1 protein, humanSurvival of Motor Neuron 1 Protein

Identifiers

PMID21482919
PMCPMC3860273
OpenAlexW2052064543

What OpenQuestion holds

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LicenceTDM
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.