MySyndrome - Rare Disease Patient Registry for Treatability, Natural History, Phenotype and Psychosozial Aspects of Rare Genetic Developmental Diseases
Recruiting
- Registration Number
- DRKS00033193
- Lead Sponsor
- kbo-Kinderzentrum / TUM Lehrstuhl Sozialpädaitrie
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 1000
Inclusion Criteria
rare genetic developmental disease
Exclusion Criteria
none
Study & Design
- Study Type
- observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 20 years after begin of the datacollection<br>The aim of the registry study is to better describe newly discovered and rare genetic developmental disorders. In particular, the effect of any off-label or approved precision therapies. In addition, the clinical course, the phenotype (especially that of the mildly or atypically affected patients) as well as psychosocial aspects of the patients and their families will be investigated.
- Secondary Outcome Measures
Name Time Method