Search for an orphan drug
Other search option(s)
Lentiviral vector containing the human liver and erythroid pyruvate kinase (PKLR) gene
- INN (International Nonproprietary Name) : -
- Code name : RP-L301
- Chemical name or description : -
- Type of product : Gene therapy product
- Type of production : Biotechnology
- Orphan designation :
Orphan designation - Europe
- Diseases list
- Hemolytic anemia due to red cell pyruvate kinase deficiency
- Detailed information
Additional information
Research on this medical product
Appraisal/reimbursement
The documents contained in this web site are presented for information purposes only. The material is in no way intended to replace professional medical care by a qualified specialist and should not be used as a basis for diagnosis or treatment.