2021
The ZSEER is a clinically and scientifically very active center. In the following year, the focus will be on expanding public relations work and thus improving visibility. This was only possible to a limited extent in 2021 due to the pandemic.
The B centers involved are striving to achieve the following goals:
- Completion of clinical trials on SARS-CoV-2 infections in children and adolescents with hypohidrotic ectodermal dysplasia and on family planning in women with XLHED.
- Establishment of an interdisciplinary Fabry center
- Elaboration of a supra-regional training structure for chronic intestinal failure
- Expansion of experimental-scientific research structures with preparation of research proposals
- Expansion of internal cooperation between the Children's Hospital and Medicine 3
- Expansion of the gene therapy center
- Expansion of the SMA screening center
- Illumination of the Erlanger Stadtwerketurm on Rare Disease Day 2022
- Maintaining the good cooperation with external centers and colleagues in private practice
- Expansion of the organizational structures in the transition area
- Expansion of supraregional research activities
- Expansion of organizational structures and networking with external experts in the context of participation in the ERN ITHACA
- Focus on the Phase 2 clinical trial started in November 2021
- Cooperation in the national networks for rare diseases being established in the diseases treated by ZSI
- Organization of a Huntington's disease information day for affected persons and their relatives
- Bavarian Genomes project; networking of centers for rare diseases in Bavaria
- Start of Phase II study Vibrant-HD
- Organization of an educational event for eosinophilic esophagitis for parents in cooperation with the Allergy Center of the UK Erlangen. The foundation of a parents' initiative is also to be initiated here. Time: September/October 2022
- Networking with external muscle centers and participation in consensus guidelines on the use of advanced therapy medicinal products (ATMPs).
- Recruit additional patients into the Bavarian Genomes project.
- Resumption of the Transition CED continuing education circle.
- Central participation in the development of an SOP for MRI imaging in muscle disease.
