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New Research Report - Bringing Down the Barriers. Updated Feb 5, 2010
From the little village of Breitnau in the Black Forest, our old friend Guenter Scheuerbrandt sends us his new report on the TREAT-NMD/NIH international conference “Bringing down the Barriers” which took place in Brussels, 17 to 19 November 2009. It is not a usual Duchenne research report, but one on the problems, “the barriers”, to be overcome if new and promising therapies and treatments are to be rapidly translated into the clinic, that is, to be made available as fast as possible to the patients. And not only Duchenne dystrophy was discussed at the meeting but a number of other neuromuscular diseases also. As before, any comments and suggestions for improvements you may have are very welcome.
Doc Scheuerbrandt's next Duchenne research report will be an update of the second part of his 2008-report about gene transfer, stem cells, utrophin, PTC124, myostatin, and other pharmacological approaches. It is due to be released at the end of April. You can read earlier reports and interviews on www.duchenne-information.eu where this new report will also be in about two weeks. And you can access some of these reports in our lefthand sidebar menu.
Dr Scheuerbrandt will be 80 in April and advises that he will probably write only one general update per year, with some small special news in between if something important happens. He will continue to answer all e-mails. Read the report...
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Research Approaches for a Therapy of Duchenne Muscular Dystrophy. Updated Nov 28, 2008
In his research reports, Dr. Guenter Scheuerbrandt, explains how the scientists and clinicians in many laboratories are trying to find effective and safe treatments for boys and young men who have Duchenne muscular dystrophy. These reports are written for them and their families and care givers in easy language so that those, who are not at home in biochemistry and medicine, will also understand what the researchers are doing for them and how far they have progressed.
The most recent report "Research Approaches" together with some earlier reports and interviews in English, German, and Spanish can be seen on the internet at www.duchenne-information.eu and can be downloaded as pdf files. This rather detailed report will be updated repeatedly with information based on presentations at scientific conferences as well as on publications in the scientific literature and on personal correspondence and discussions with many scientists. Scan current report and archives...
Exon Skipping, a therapy for Duchenne muscular dystrophy. Updated September, 2008
Interview with Dr. Gerard Platenburg, President of Prosensa B.V. Leiden, the Netherlands.
This interview was recorded in Philadelphia by me, Guenter Scheuerbrandt PhD., on 19 July 2008 at the annual meeting ofPPMD, the American Parent Project Muscular Dystrophy. The following text is an edited and shortened version of the spoken interview. It has been approved by Dr. Platenburg for the information of patients, their families, and care-givers. My questions are written in italics, the answers of Dr. Platenburg in normal print. The chapters about the clinical trial in the Netherlands, as they appeared in my last report "Research approaches for a therapy of Duchenne muscular dystrophy" are shown at the end of this text and should be read as an introduction to this interview. Read the full-text interview
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Reading through premature stop codons with PTC124. Project Catalyst to find more Duchenne drugs.
Interview with Ellen Welch PhD., Diane Goetz, and Neil Almstead PhD. Updated September, 2008
This interview was recorded at the company PTC Therapeutics in South Plainfield, New Jersey (about 20 miles west of New York) by me, Guenter Scheuerbrandt PhD., on 21 July 2008 after the annual meeting of PPMD, the American Parent Project Muscular Dystrophy, in Philadelphia. The following text is an edited and shortened version of the original interview. It has been approved by PTC for the information of patients, their families, and care-givers. My questions are written in italics, the answers of Ellen Welch, Associate Director, Genetic Disorders; Diane Goetz, Director, Patient and Professional Advocacy; and Neil Almstead, Senior Vice President, Chemistry, in normal print. The chapters about PTC124 and Project Catalyst, as they appeared in my last report "Research approaches for a therapy of Duchenne muscular dystrophy" are shown at the end of this text and should be read as an introduction to this interview. Read the full text of interview
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Research Approaches for a Therapy of Duchenne Muscular Dystrophy. Updated in July 2008
This is a new kind of research report, which I, Günter Scheuerbrandt, a biochemist in Germany, have written for you, the Duchenne boys and young men and their families, who wish to know how the work of scientists and clinicians in many research laboratories of the world is progressing towards effective therapies for Duchenne muscular dystrophy. My earlier reports, and especially the last three on the Parent Project meetings in Cincinnati in July 2006, in London 2006, and Philadelphia 2007 (www.duchenne-information.eu), contained rather detailed summaries of the research results presented at these meetings. But as also other important scientific research is performed without having yet been mentioned at the meetings, I have now shortened practically all summaries of the three last reports for this report, updated them with new information, mainly from ActionDuchenne's meeting in London in November 2007, and have added new summaries of recent important publications. Ref-erences to some of the most important publications are given at the end of some summaries.
This is now a basic text, which is up-to-date as of July 2008. I will update it repeatedly with new information, the first time after the next Parent Project meeting in Philadelphia in July 2008; it will be available in English, Spanish, and German a few months later. As before, all my reports and this one, too, are not scientific publications with many difficult words, because I have tried to write it in a way that will let you understand what is happening for you in the laboratories. Read more...
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Read Dr Scheuerbrandt's full report from the PPMD Philidelphia Conference 12 - 14 July 2007. You will find the report in other places: www.duchenne-information.eu and on the PPMD website, who commissioned the report www.parentprojectmd.org
As always this is a lucid report written especially for the layperson and Doc Guenter has analysed the new research presented at the British conference in November, 2007 and the report has been updated. This is a wonderful service for parents, provided by a wonderful professional. Thank you Dr Scheuerbrandt.
During the year, many e-mails from all over the world reach Dr Scheuerbrandt, especially from families with Duchenne boys, in which questions were asked mainly about research for a therapy of Duchenne muscular dystrophy. As it is impossible for him to answer all questions individually, he has written a general question-and-answers text [Below]
English Version
German Version
Spanish Version
This text has been distributed not only to those who have sent the questions, but to all families and patients of his acquaintance, because his answers will probably interest all Duchenne families and many researchers and clinical experts (whom he invites to comment and suggest any changes or additions.)
Our good friend Dr Guenter Scheuerbrandt, PhD. lives in Breitnau in the Black Forest in Germany.
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