Prader-Willi Syndrome
An autosomal dominant disorder caused by deletion of the proximal long arm of the paternal chromosome 15 (15q11-q13) or by inheritance of both of the pair of chromosomes 15 from the mother (UNIPARENTAL DISOMY) which are imprinted (GENETIC IMPRINTING) and hence silenced. Clinical manifestations include MENTAL RETARDATION; MUSCULAR HYPOTONIA; HYPERPHAGIA; OBESITY; short stature; HYPOGONADISM; STRABISMUS; and HYPERSOMNOLENCE. (Menkes, Textbook of Child Neurology, 5th ed, p229)
Drugs by status
Approved for this indication (2)
Phase 4 trials (4)
Phase 3 trials (12)
Phase 2 trials (9)
Phase 1 trials (1)
Organizations
Organization Involved with Phase 4 Indications (8)
Organization Involved with Phase 3 Indications (23)
- Pfizer
- LG
- Millendo Therapeutics, Inc.
- Zafgen, Inc.
- GeneScience Pharmaceuticals Co., Ltd.
- National Institutes of Health (NIH)
- Huazhong University of Science and Technology
- Cornell University
- University of Kansas
- Capital Medical University
- Epidemiological and Clinical Research Information Network
- Fudan University
- Peking University
- Zhejiang University
- University of Paris
- Erasmus University Rotterdam
- Foundation for Prader-Willi Research
- International Clinical Trials Association
- Prader Willi Syndrome Association
- Soleno Therapeutics, Inc.
- Chinese Academy of Medical Sciences
- Levo Therapeutics, Inc.
- Prader-Willi Fonds
Organization Involved with Phase 2 Indications (11)
Organization Involved with Other Experimental Indications (8)