Rare disease patients get early access to promising treatment
NCT ID NCT05911399
First seen Nov 01, 2025 · Last updated Apr 29, 2026 · Updated 27 times
Summary
This program offers early access to tiratricol for people with Allan-Herndon-Dudley syndrome, a rare genetic disorder that affects movement and development. Participants must have a confirmed genetic diagnosis and may be new to tiratricol or already taking it from a previous study. The goal is to provide treatment before official approval, based on the doctor's judgment that the benefits outweigh the risks.
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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.
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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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
AVAILABLEChicago, Illinois, 60611, United States
Contact
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Baystate Medical Center
AVAILABLESpringfield, Massachusetts, 01199, United States
Contact
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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
Contact
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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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Icahn School of Medicine at Mount Sinai
AVAILABLENew York, New York, 10029, 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.