| Item | Content |
|---|---|
| Research project name | Practical Research Project for Rare / Intractable Diseases |
| Study title | Development of drugs containing medium chain fatty acid for triglyceride deposit cardiomyovasculopathy |
| Name of principal investigator | Kenichi Hirano |
| Name of institution of the principal investigator | Department of Cardiovascular Medicine, Faculty of Internal Medicine, Graduate School of Medicine, Osaka University |
| Target disease | Neutral lipid storage disease (NLSD), triglyceride deposit cardiomyovasculopathy (TGCV) |
| Registry name | International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related Diseases |
| Purpose of registry | Survey of natural history, Investigation into the number or the distribution of patients, Epidemiological study, Patient recruitment for clinical trials or other interventional studies |
| Overview of registry | The objectives of this study are collection of epidemiological information on neutral lipid storage disease and triglyceride deposit cardiomyovasculopathy (e.g., patient distribution, prognosis, prognostic factors, disease type) and recruitment of subjects for the clinical study. |
| Research item | Sex, Date of birth, Place of birth, Family history, Complication, History of present illness, Laboratory data, Others (presence of gene mutations, date of diagnosis/onset, presence of medication or treatment) |
| Contact | Akira Suzuki |
| E-mail address | registry●cnt-osaka.com |
| Registry URL | http://www.tgcv.org/r/home.html |
* If the E-mail address is shown, replace "●" with "@", please.