RIBOMIC Announces Dosing of First Subject in RBM-007 Phase 1 Clinical Trial for Achondroplasia
Retrieved on:
Wednesday, July 15, 2020
Health, Genetics, Clinical trials, Research, Pharmaceutical, Science, Branches of biology, Growth disorders, Tyrosine kinase receptors, Rare diseases, Fibroblast growth factor receptor 3, Health, Medicine, Achondroplasia, Fibroblast growth factor receptor, Fibroblast growth factor, Hypochondroplasia, Achondroplasia in children, Phase 1 study, Achondroplasia
Achondroplasia has no known cure, and is designated as an intractable disease by the Ministry of Health, Labour and Welfare in Japan.
Key Points:
- Achondroplasia has no known cure, and is designated as an intractable disease by the Ministry of Health, Labour and Welfare in Japan.
- This disease results mainly from a genetic variant in FGFR3 (fibroblast growth factor type 3 receptor).
- Achondroplasia occurs in a frequency of 1 in approximately 25,000 normal live births and is estimated to affect approximately 250,000 people worldwide.
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