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Rett Syndrome
An inherited neurological developmental disorder that is associated with X-LINKED INHERITANCE and may be lethal in utero to hemizygous males. The affected female is normal until the age of 6-25 months when progressive loss of voluntary control of hand movements and communication skills; ATAXIA; SEIZURES; autistic behavior; intermittent HYPERVENTILATION; and HYPERAMMONEMIA appear. (From Menkes, Textbook of Child Neurology, 5th ed, p199)
Drugs by status
Approved for this indication (1)
Phase 2 trials (15)
Phase 1 trials (2)
Organizations
Organization Involved with Phase 3 Indications (8)
Organization Involved with Phase 2 Indications (25)
- Novartis
- Bioelectron
- Neuren Pharmaceuticals Limited
- University of Pennsylvania
- Baylor University
- Harvard University
- Emory University
- Gillette Children's Specialty Healthcare
- Johns Hopkins University
- University of Bern
- Tel Aviv University
- University of California, San Diego
- University of California, San Francisco
- University of Cincinnati
- Vanderbilt University
- University of Basel
- Greenwood Genetic Center
- Ultragenyx Inc.
- Rush University
- University of Alabama, Birmingham
- University of Colorado
- Aix-University of Marseille
- Rett Syndrome Research Trust
- International Rett Syndrome Foundation
- Yeshiva University
Organization Involved with Phase 1 Indications (1)
Organization Involved with Other Experimental Indications (3)