Literature DB >> 26250347

Review of Spinal Muscular Atrophy (SMA) for Prenatal and Pediatric Genetic Counselors.

Amanda Carré1, Candice Empey2.   

Abstract

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular condition with degeneration of the anterior horn cells in the spinal column. Five SMA subtypes exist with classification dependent upon the motor milestones achieved. Study of the SMN1 (survival motor neuron) and SMN2 genes as well as the concepts of the "2 + 0" carriers, gene conversion, de novo mutations and intragenic mutations allow for a better understanding of SMA. Detailing the carrier and diagnostic testing options further deepens the genetic counselor's knowledge of SMA. A review of care guidelines and research options is included as this information gives a patient a well-rounded view of SMA. Although SMA is most commonly associated with the SMN1 gene, a number of spinal muscular atrophies not caused by genetic changes in this gene may be included as differential diagnoses until confirmatory testing can be completed. SMA is a complex condition requiring a detailed knowledge on the genetic counselor's part in order to explain the disorder to the patient with clarity thus facilitating increased communication and decision making guidance with the patient.

Entities:  

Keywords:  Carrier screening; Diagnostic testing; Genetic counseling; Pediatric; Prenatal; Spinal muscular atrophy

Mesh:

Substances:

Year:  2015        PMID: 26250347     DOI: 10.1007/s10897-015-9859-z

Source DB:  PubMed          Journal:  J Genet Couns        ISSN: 1059-7700            Impact factor:   2.537


  67 in total

1.  Identification of proximal spinal muscular atrophy carriers and patients by analysis of SMNT and SMNC gene copy number.

Authors:  P E McAndrew; D W Parsons; L R Simard; C Rochette; P N Ray; J R Mendell; T W Prior; A H Burghes
Journal:  Am J Hum Genet       Date:  1997-06       Impact factor: 11.025

Review 2.  Genetic testing and risk assessment for spinal muscular atrophy (SMA).

Authors:  Shuji Ogino; Robert B Wilson
Journal:  Hum Genet       Date:  2002-10-03       Impact factor: 4.132

Review 3.  An update of the mutation spectrum of the survival motor neuron gene (SMN1) in autosomal recessive spinal muscular atrophy (SMA).

Authors:  B Wirth
Journal:  Hum Mutat       Date:  2000       Impact factor: 4.878

4.  Consensus statement for standard of care in spinal muscular atrophy.

Authors:  Ching H Wang; Richard S Finkel; Enrico S Bertini; Mary Schroth; Anita Simonds; Brenda Wong; Annie Aloysius; Leslie Morrison; Marion Main; Thomas O Crawford; Anthony Trela
Journal:  J Child Neurol       Date:  2007-08       Impact factor: 1.987

5.  SMA CARNI-VAL trial part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy.

Authors:  Kathryn J Swoboda; Charles B Scott; Thomas O Crawford; Louise R Simard; Sandra P Reyna; Kristin J Krosschell; Gyula Acsadi; Bakri Elsheik; Mary K Schroth; Guy D'Anjou; Bernard LaSalle; Thomas W Prior; Susan L Sorenson; Jo Anne Maczulski; Mark B Bromberg; Gary M Chan; John T Kissel
Journal:  PLoS One       Date:  2010-08-19       Impact factor: 3.240

6.  Homozygous SMN1 deletions in unaffected family members and modification of the phenotype by SMN2.

Authors:  Thomas W Prior; Kathryn J Swoboda; H Denman Scott; Ashley Q Hejmanowski
Journal:  Am J Med Genet A       Date:  2004-10-15       Impact factor: 2.802

7.  Treatment of type I spinal muscular atrophy with noninvasive ventilation and gastrostomy feeding.

Authors:  D J Birnkrant; J F Pope; J E Martin; A H Repucci; R M Eiben
Journal:  Pediatr Neurol       Date:  1998-05       Impact factor: 3.372

8.  Deletions in the survival motor neuron gene on 5q13 in autosomal recessive spinal muscular atrophy.

Authors:  N R Rodrigues; N Owen; K Talbot; J Ignatius; V Dubowitz; K E Davies
Journal:  Hum Mol Genet       Date:  1995-04       Impact factor: 6.150

9.  Hexosaminidase A deficiency manifesting as spinal muscular atrophy of late onset.

Authors:  A Karni; R Navon; M Sadeh
Journal:  Ann Neurol       Date:  1988-09       Impact factor: 10.422

Review 10.  Therapy development for spinal muscular atrophy in SMN independent targets.

Authors:  Li-Kai Tsai
Journal:  Neural Plast       Date:  2012-05-31       Impact factor: 3.599

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  4 in total

Review 1.  A Review on Spinal Muscular Atrophy: Awareness, Knowledge, and Attitudes.

Authors:  Rebecca R Moultrie; Julia Kish-Doto; Holly Peay; Megan A Lewis
Journal:  J Genet Couns       Date:  2016-04-16       Impact factor: 2.537

2.  ML372 blocks SMN ubiquitination and improves spinal muscular atrophy pathology in mice.

Authors:  Mahlet B Abera; Jingbo Xiao; Jonathan Nofziger; Steve Titus; Noel Southall; Wei Zheng; Kasey E Moritz; Marc Ferrer; Jonathan J Cherry; Elliot J Androphy; Amy Wang; Xin Xu; Christopher Austin; Kenneth H Fischbeck; Juan J Marugan; Barrington G Burnett
Journal:  JCI Insight       Date:  2016-11-17

3.  An Evidence-Based, Community-Engaged Approach to Develop an Interactive Deliberation Tool for Pediatric Neuromuscular Trials.

Authors:  Rebecca R Moultrie; Megan A Lewis; Ryan S Paquin; Ann Lucas; Jill Jarecki; Holly L Peay
Journal:  J Genet Couns       Date:  2017-12-20       Impact factor: 2.537

4.  Detection of Spinal Muscular Atrophy Patients Using Dried Saliva Spots.

Authors:  Yogik Onky Silvana Wijaya; Hisahide Nishio; Emma Tabe Eko Niba; Kentaro Okamoto; Haruo Shintaku; Yasuhiro Takeshima; Toshio Saito; Masakazu Shinohara; Hiroyuki Awano
Journal:  Genes (Basel)       Date:  2021-10-14       Impact factor: 4.096

  4 in total

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