Literature DB >> 20128138

The progress reports on the development of therapies of Duchenne muscular dystrophy.

G Scheuerbrandt1.   

Abstract

The roots of the progress reports on the development of therapies for Duchenne muscular dystrophy (DMD) that since 2000 have been produced at Breitnau/Germany and distributed to the parents of DMD patients cover over 30 years of continual occupation with this disease. The beginning was marked by the development of an early detection programme for the genetic disposition for DMD in infant boys. The next step was the organisation of workshops on the management of DMD and the writing of progress reports on these and other relevant conferences. Getting acquainted with the ideas of the protagonists in the research field by holding interviews was a decisive prerequisite for this activity. This took place in tandem with the development of a new kind of multiplex "family letters" that attempted to answer frequently asked questions to many DMD families at the same time. When--with the beginning of the new millennium--the endeavours towards gene therapies for DMD started to boom all over the scientific world, progress reports designed to keep the families informed about research on DMD treatment were added to the family letters. These reports that give an account of the latest state of the research are written in a plain language that can be understood by laypersons. In the meantime the reports have adopted the character of reviews that are updated annually. They are written in English and German and translated into Spanish and many other languages.

Entities:  

Mesh:

Year:  2009        PMID: 20128138      PMCID: PMC2858951     

Source DB:  PubMed          Journal:  Acta Myol        ISSN: 1128-2460


  6 in total

1.  Approaching therapies for boys with Duchenne muscular dystrophy. Parent Project Muscular Dystrophy. Annual conference in Cincinnati/Ohio, 13-16 July 2006.

Authors:  Günter Scheuerbrandt
Journal:  Acta Myol       Date:  2006-10

Review 2.  Neonatal screening for muscular dystrophy. Consensus recommendation of the 14th workshop sponsored by the European Neuromuscular Center (ENMC).

Authors:  G J van Ommen; G Scheuerbrandt
Journal:  Neuromuscul Disord       Date:  1993-05       Impact factor: 4.296

3.  Newborn screening for Duchenne muscular dystrophy.

Authors:  H Zellweger; A Antonik
Journal:  Pediatrics       Date:  1975-01       Impact factor: 7.124

4.  Screening for Duchenne muscular dystrophy: an improved screening test for creatine kinase and its application in an infant screening program.

Authors:  G Scheuerbrandt; A Lundin; T Lövgren; W Mortier
Journal:  Muscle Nerve       Date:  1986-01       Impact factor: 3.217

5.  First meeting of the Duchenne Parent Project in Europe: Treatment of Duchenne Muscular Dystrophy. 7-8 November 1997, Rotterdam, The Netherlands.

Authors:  G Scheuerbrandt
Journal:  Neuromuscul Disord       Date:  1998-05       Impact factor: 4.296

Review 6.  Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations.

Authors:  Annemieke Aartsma-Rus; Ivo Fokkema; Jan Verschuuren; Ieke Ginjaar; Judith van Deutekom; Gert-Jan van Ommen; Johan T den Dunnen
Journal:  Hum Mutat       Date:  2009-03       Impact factor: 4.878

  6 in total

北京卡尤迪生物科技股份有限公司 © 2022-2023.