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Newsletter May 2013

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News: EMA Consultation: Draft guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy
The European Medicines Agency has recently published a draft guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy. Recent advances in basic and clinical research have opened new perspectives for future therapeutic options in Duchenne and Becker muscular dystrophy (DBMD). As most of the cases of Duchenne muscular dystrophy (DMD) have an onset in early childhood, while the onset of Becker muscular dystrophy (BMD) covers a broader age spectrum, specific difficulties have been identified that pertain to diagnostic criteria, age- and stage related clinical relevance and different safety aspects.

News: Netherlands Gene Therapy Office launches website
The aim of the governmental Gene Therapy Office is to streamline the licensing and permit granting procedures for clinical gene therapy studies in The Netherlands and to provide more insight into the procedures for investigators. The Gene Therapy Office is primarily a service for professionals conducting clinical gene therapy research involving human subjects. The official website is

- 15 – 18 May 2013, American Society of Gene and Cell Therapy (ASGCT) 16th Annual Meeting, Salt Lake City, Utah
- 4 – 6 July 2013, The 19th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Okayama, Japan
- 12 – 14 August 2013, International Conference on Genetic Engineering & Genetically Modified Organisms, Raleigh, North Carolina
- 23 – 25 October 2013, 2nd International Conference and Exhibition on Cell & Gene Therapy, Orlanda, Florida
- 24 – 28 October 2013, VII Congress of the Spanish Society of Gene (SETGyC) and Cell Therapy, and XXI Congress of the European Society of Gene and Cell Therapy (ESGCT), Madrid, Spain
- 20 – 22 November 2013, 12th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases, Sonoma, California


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