Experimental drug offered to patients with rare, severe genetic disorder
NCT ID NCT05911399
Summary
This program provides access to an experimental drug called tiratricol for patients with Allan-Herndon-Dudley syndrome, a rare and severe genetic disorder that affects brain development and movement. Eligible patients who cannot join clinical trials can receive this treatment before it's officially approved. Doctors will monitor patients regularly to check how they respond and watch for any side effects.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Email: •••••@•••••
Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
AVAILABLEChicago, Illinois, 60611, United States
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Baystate Medical Center
AVAILABLESpringfield, Massachusetts, 01199, United States
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Children's Healthcare of Atlanta
AVAILABLEAtlanta, Georgia, 30329, United States
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Children's Hospital of Philadelphia
AVAILABLEPhiladelphia, Pennsylvania, 19104, United States
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Children's Wisconsin
AVAILABLEMilwaukee, Wisconsin, 53326, United States
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Columbia University Irving Medical Center
AVAILABLENew York, New York, 10032, United States
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Cook Children's Health Care System
AVAILABLEFort Worth, Texas, 76106, United States
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Gillette's Children's Specialty Healthcare
AVAILABLESaint Paul, Minnesota, 55101, United States
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Le Bonheur Children's Hospital Foundation
AVAILABLEMemphis, Tennessee, 38105, United States
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Louisiana State University Health Sciences Center and Children's Hospital
AVAILABLENew Orleans, Louisiana, 70118, United States
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MultiCare Mary Bridge Children's Hospital
AVAILABLETacoma, Washington, 98403, United States
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Nemour's Children Hospital
AVAILABLEJacksonville, Florida, 32207, United States
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Oregon Health and Science University
AVAILABLEPortland, Oregon, 97239, United States
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Rady Children's Hospital
AVAILABLESan Diego, California, 92123, United States
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University of Alabama at Birmingham Hospital
AVAILABLEBirmingham, Alabama, 35249, United States
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Valley Children's Hospital - Enrollment limited to patients within the hospital's health system
AVAILABLEMadera, California, 93636-8762, United States
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Conditions
Explore the condition pages connected to this study.