Methods for treating allan-herndon-dudley syndrome
Inventors
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Assignees
Dexcel Pharma Technologies Ltd • University of Chicago
MemberUniversity of MiamiUniversity of MiamiThe University of Miami, established in 1925 and based in Coral Gables, Florida, is a private research university recognized for its comprehensive academic offerings, robust research infrastructure, and strong interdisciplinary focus. Home to more than 19,000 students and with more than 400 acres of campuses across the Miami region, its mission encompasses education, research, innovation, and community service. The institution supports clinical, biomedical, marine, and atmospheric research initiatives, delivers diverse undergraduate and graduate programs, and maintains numerous research centers and institutes dedicated to scientific, medical, and societal advancements.
The University of Miami, established in 1925 and based in Coral Gables, Florida, is a private research university recognized for its comprehensive academic offerings, robust research infrastructure, and strong interdisciplinary focus. Home to more than 19,000 students and with more than 400 acres of campuses across the Miami region, its mission encompasses education, research, innovation, and community service. The institution supports clinical, biomedical, marine, and atmospheric research initiatives, delivers diverse undergraduate and graduate programs, and maintains numerous research centers and institutes dedicated to scientific, medical, and societal advancements.
Publication Number
US-12390432-B2
Publication Date
2025-08-19
Expiration Date
Abstract
The present subject matter is directed to methods of treating Allan-Herndon-Dudley syndrome comprising administering 3,5-diiodothyropropionic acid (DITPA) to a subject in need thereof, wherein the DITPA administration reduces triiodothyronine (“T3”) serum levels to normal, increases T3 brain levels to normal, and maintains normal serum levels of thyroxine (T4) and thyroid stimulating hormone (TSH). The subject may be a child or an adult.
Core Innovation
The invention relates to a method of treating Allan-Herndon-Dudley syndrome by administering 3,5-diiodothyropropionic acid (DITPA) to a subject in need thereof. Treatment begins within three days after birth and uses a total daily dosage of at least 2 milligrams per kilogram of body weight.
The method aims to reduce triiodothyronine (T3) serum levels to normal and increase T3 brain levels to normal. The method further aims to increase to, or maintain, normal thyroxine (T4) serum levels and to decrease to, or maintain, normal thyroid stimulating hormone (TSH) serum levels.
The disclosed approach includes staged or step-up dosing regimens in which a first daily dosage is followed by a second daily dosage for additional periods. The staged dosing is associated with T3-serum-guided continuation or adjustment, including increasing or decreasing the daily dosage when T3 is too high or too low.
Claims Coverage
The partial content explicitly provides two independent claims directed to DITPA-based treatment of Allan-Herndon-Dudley syndrome, with core inventive features centered on timing after birth, daily dosage magnitude, and biomarker normalization targets, and with a step-up two-period regimen in the second independent claim.
DITPA administration within three days after birth for AHDS
Administration begins within three days after birth of the subject.
At least 2 mg/kg/day total daily DITPA dose for AHDS treatment
Administration is in a total daily dosage of at least 2 milligrams per kilogram of body weight of the subject.
Biomarker normalization of T3 serum, T3 brain, T4, and TSH
Administration reduces triiodothyronine (T3) serum levels to normal, increases T3 brain levels to normal, increases to, or maintains, normal serum levels of thyroxine (T4), and decreases to, or maintains, normal thyroid stimulating hormone (TSH) serum levels.
Two-week first and second daily DITPA dosages with step-up
Daily DITPA is administered for two weeks at a first daily dosage, then for two weeks at a second daily dosage greater than the first.
First daily DITPA dosage of at least 2 mg/kg within a two-week period
Step a) administers DITPA daily at a first daily dosage, of at least 2 milligrams per kilogram of body weight, for two weeks.
Across the two independent claims, the core claim coverage is the use of DITPA for treating Allan-Herndon-Dudley syndrome, initiated within three days after birth, using at least 2 mg/kg/day total daily dosage, and targeting normalization of T3 serum, T3 brain, T4, and TSH, with the second independent claim further requiring a two-period two-week regimen that increases the daily dosage from a first dose to a greater second dose.
Stated Advantages
Reduces triiodothyronine (T3) serum levels to normal.
Increases T3 brain levels to normal.
Increases to, or maintains, normal thyroxine (T4) serum levels.
Decreases to, or maintains, normal thyroid stimulating hormone (TSH) serum levels.
Documented Applications
Treatment of Allan-Herndon-Dudley syndrome (AHDS/ARDS) in a subject in need thereof using DITPA.
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