Article
Danazol Treatment for Telomere Diseases.
The New England journal of medicine - 19 May 2016
Townsley Danielle M, Dumitriu Bogdan, Liu Delong, Biancotto Angélique, Weinstein Barbara, Chen Christina, Hardy Nathan, Mihalek Andrew D, Lingala Shilpa, Kim Yun Ju, Yao Jianhua, Jones Elizabeth, Gochuico Bernadette R, Heller Theo, Wu Colin O, Calado Rodrigo T, Scheinberg Phillip, Young Neal S
Abstract excerpt
BACKGROUND: Genetic defects in telomere maintenance and repair cause bone marrow failure, liver cirrhosis, and pulmonary fibrosis, and they increase susceptibility to cancer. Historically, androgens have been useful as treatment for marrow failure syndromes. In tissue culture and animal models, sex hormones regulate expression of the telomerase gene. METHODS: In a phase 1-2 prospective study involving patients...
Topics
- Administration, Oral
- Adolescent
- Adult
- Aged
- Bone Marrow Diseases
- Danazol
- Estrogen Antagonists
- Female
- Hair Color
- Humans