Michael Beck – JOGU – Johannes Gutenberg University, Mainz, Germany

michaelbeckInstitute description
The division of Lysosomal Storage Disorders at the Children’s Hospital, University of Mainz takes care of more than 500 patients with lysosomal storage disorders. This institution has fifteen years of experience in enzyme replacement therapy in LSDs. Scientific work includes clinical studies in Fabry disease, Gaucher disease, Mucopolysaccharidoses and Glycoprotein storage disorders. Almost 200 Gaucher patients, more than 100 Fabry patients, 20 Pompe patients and about 60 MPS patients receive an enzyme replacement therapy in the Institute. A laboratory for the diagnosis of all lysosomal storage disorders is available in the department.

Group leader
Michael Beck, is Head of the centre for Lysomal Storage Disorders at the Children’s Hospital of the University of Mainz since 2001 and takes care of over 400 patients with various LSDs. Presently, Dr Beck is principal investigator in several clinical trials (enzyme replacement therapy in Lysosomal Storage Disorders).

Dr Beck received his pediatric training at the Department of Pediatrics, University of Frankfurt. His special interests were endocrine disorders and lysosomal storage diseases. Training in genetics was received at the Institute of Human Genetics in Frankfurt, where he worked for one year (1979-1980). Here he was introduced to genetic counseling and diagnosis of malformation syndromes. Since October 1980, he is working at the Department of Pediatrics of the University of Mainz. Furthermore, Dr Beck is member of the Deutsche Gesellschaft für Kinderheilkunde, Deutsche Gesellschaft für Humangenetik, SSIEM (Society for the Study of Inborn Errors of Metabolism), ESPR (European Society for Pediatric Research), the ESGLD (European Study Group on Lysosomal Diseases) and American Society of Human Genetics (ASHG).

Role in the project
The expertise of Dr Beck and his staff is relevant for the development of WP2.
The centre for Lysomal Storage Disorders in Mainz has all the qualifications to perform clinical tests (lung function, echocardiography, electrocardiogram, orthopedic tests etc.) since it has participated at several clinical trials. In addition to physicians who are trained in clinical trials, experienced study nurses are also part of the staff. The centre for Lysosomal Storage Disorders is located in a new facility that is solely destined to clinical studies.

Key personnel

Name Title Gender Role/Expertise
Michael Beck Group leader M Involved in WP2
Eugen Mengel Investigator M ???

Key publications

Beck M (2007). New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy. Hum Genet 121: 1-22.

Muenzer J, Wraith JE, Beck M, Giugliani R, Harmatz P, Eng CM, Vellodi A, Martin R, Ramaswami U, Gucsavas-Calikoglu M, Vijayaraghavan S, Wendt S, Puga AC, Ulbrich B, Shinawi M, Cleary M, Piper D, Conway AM, Kimura A (2006). A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet Med 8: 465-473.

Beck M (2006). Galsulfase: enzyme-replacement therapy for mucopolysaccharidosis Type VI (Maroteaux-Lamy syndrome). Therapy 3: 9-17.

Baehner F, Schmiedeskamp C, Krummenauer F, Miebach E, Bajbouj M, Whybra C, Kohlschutter A, Kampmann C, Beck M (2005). Cumulative incidence rates of the mucopolysaccharidoses in Germany. J Inherit Metabol Dis 28: 1011-1017.