Literature DB >> 26260293

Twenty-year follow-up of newborn screening for patients with muscular dystrophy.

Jeffrey Chung1, Andrea L Smith1, Sarah C Hughes1, Gabriela Niizawa1, Hoda Z Abdel-Hamid2, Edwin W Naylor3, Timothy Hughes4, Paula R Clemens1,5.   

Abstract

INTRODUCTION: An opt-out newborn screening (NBS) program for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) was implemented at 2 hospitals in Pittsburgh, Pennsylvania, between 1987 and 1995.
METHODS: For patients and their parents in families who received a diagnosis of DMD or BMD, either by NBS or by traditional diagnostics after symptom onset, attitudes toward NBS for DMD and BMD were assessed.
RESULTS: All patients and most parents supported NBS for DMD and BMD. In contrast to the NBS parent cohort, the non-NBS cohort felt that diagnosis by NBS would cause anxiety.
CONCLUSIONS: There was strong support of NBS for DMD and BMD in both patients and their parents in families who received a diagnosis through NBS or through traditional diagnostics. No negative psychosocial impacts of NBS were identified among those families who received a diagnosis through NBS.
© 2015 Wiley Periodicals, Inc.

Entities:  

Keywords:  Becker muscular dystrophy; Duchenne muscular dystrophy; family planning; newborn screening; parental attitudes; patient attitudes; public health policy

Mesh:

Year:  2015        PMID: 26260293     DOI: 10.1002/mus.24880

Source DB:  PubMed          Journal:  Muscle Nerve        ISSN: 0148-639X            Impact factor:   3.217


  11 in total

Review 1.  Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management.

Authors:  David J Birnkrant; Katharine Bushby; Carla M Bann; Susan D Apkon; Angela Blackwell; David Brumbaugh; Laura E Case; Paula R Clemens; Stasia Hadjiyannakis; Shree Pandya; Natalie Street; Jean Tomezsko; Kathryn R Wagner; Leanne M Ward; David R Weber
Journal:  Lancet Neurol       Date:  2018-02-03       Impact factor: 44.182

2.  In vivo restoration of dystrophin expression in mdx mice using intra-muscular and intra-arterial injections of hydrogel microsphere carriers of exon skipping antisense oligonucleotides.

Authors:  Shani Attias Cohen; Orit Bar-Am; Claudia Fuoco; Galit Saar; Cesare Gargioli; Dror Seliktar
Journal:  Cell Death Dis       Date:  2022-09-09       Impact factor: 9.685

3.  The case for screening in early life for 'non-treatable' disorders: ethics, evidence and proportionality. A report from the Health Council of the Netherlands.

Authors:  Shona Kalkman; Wybo Dondorp
Journal:  Eur J Hum Genet       Date:  2022-02-07       Impact factor: 5.351

4.  Characterizing Enrollment in Observational Studies of Duchenne Muscular Dystrophy by Race and Ethnicity.

Authors:  Alison M Barnard; Samuel L Riehl; Rebecca J Willcocks; Glenn A Walter; Amber M Angell; Krista Vandenborne
Journal:  J Neuromuscul Dis       Date:  2020

5.  Identification of critical molecular pathways involved in exosome-mediated improvement of cardiac function in a mouse model of muscular dystrophy.

Authors:  Xuan Su; Yan Shen; Yue Jin; Neal L Weintraub; Yao-Liang Tang
Journal:  Acta Pharmacol Sin       Date:  2020-06-29       Impact factor: 6.150

6.  Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy.

Authors:  Nathalie Goemans; Marleen Vanden Hauwe; James Signorovitch; Elyse Swallow; Jinlin Song
Journal:  PLoS One       Date:  2016-10-13       Impact factor: 3.240

7.  Diagnosis of Duchenne Muscular Dystrophy in a Presymptomatic Infant Using Next-Generation Sequencing and Chromosomal Microarray Analysis: A Case Report.

Authors:  Eun-Woo Park; Ye-Jee Shim; Jung-Sook Ha; Jin-Hong Shin; Soyoung Lee; Jang-Hyuk Cho
Journal:  Children (Basel)       Date:  2021-05-11

8.  Brain and behavior development in autism from birth through infancy.

Authors:  Mark D Shen; Joseph Piven
Journal:  Dialogues Clin Neurosci       Date:  2017-12       Impact factor: 5.986

9.  Link between MHC Fiber Type and Restoration of Dystrophin Expression and Key Components of the DAPC by Tricyclo-DNA-Mediated Exon Skipping.

Authors:  Saleh Omairi; Kwan-Leong Hau; Henry Collin-Hooper; Federica Montanaro; Aurelie Goyenvalle; Luis Garcia; Ketan Patel
Journal:  Mol Ther Nucleic Acids       Date:  2017-10-26

Review 10.  Is it the right time for an infant screening for Duchenne muscular dystrophy?

Authors:  Gian Luca Vita; Giuseppe Vita
Journal:  Neurol Sci       Date:  2020-02-28       Impact factor: 3.307

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