Expanded Access Program for Tiratricol in Patients With Monocarboxylate Transporter 8 Deficiency
Condition(s):Monocarboxylate Transporter 8 Deficiency; Allan-Herndon-Dudley SyndromeLast Updated:December 13, 2023Available
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Condition(s):Monocarboxylate Transporter 8 Deficiency; Allan-Herndon-Dudley SyndromeLast Updated:December 13, 2023Available
Condition(s):Monocarboxylate Transporter 8 DeficiencyLast Updated:August 24, 2021Unknown status
Condition(s):Monocarboxylate Transporter 8 Deficiency; Allan-Herndon-Dudley SyndromeLast Updated:January 18, 2024Recruiting
Condition(s):Allan-Herndon-Dudley SyndromeLast Updated:October 31, 2022Active, not recruiting
Condition(s):Allan-Herndon-Dudley SyndromeLast Updated:April 16, 2019Completed
Condition(s):Monocarboxylate Transporter 8 (MCT8) Deficiency; Allan-Herndon-Dudley SyndromeLast Updated:January 18, 2024Recruiting
Condition(s):Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter DeficiencyLast Updated:November 18, 2023Available
Condition(s):Thyroid MetabolismLast Updated:May 3, 2017Completed
Condition(s):Leukodystrophy; White Matter Disease; Leukoencephalopathies; 4H Syndrome; Adrenoleukodystrophy; AMN; ALD; ALD Gene Mutation; ALD (Adrenoleukodystrophy); X-linked Adrenoleukodystrophy; X-ALD; Adrenomyeloneuropathy; Aicardi Goutieres Syndrome; AGS; Alexander Disease; Alexanders Leukodystrophy; AxD; ADLD; Canavan Disease; CTX; Cerebrotendinous Xanthomatoses; Krabbe Disease; GALC Deficiency; Globoid Leukodystrophy; TUBB4A-Related Leukodystrophy; H-ABC – Hypomyelination, Atrophy of Basal Ganglia and Cerebellum; HBSL; HBSL – Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity; LBSL; Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder); Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation; ALSP; CSF1R Gene Mutation; HCC – Hypomyelination and Congenital Cataract; MLC1; Megalencephalic Leukoencephalopathy With Subcortical Cysts; MLD; Metachromatic Leukodystrophy; PMD; Pelizaeus-Merzbacher Disease; PLP1 Null Syndrome; PLP1 Gene Duplication | Blood or Tissue | Mutations; Pelizaeus Merzbacher Like Disease; Peroxisomal Biogenesis Disorder; Zellweger Syndrome; Refsum Disease; Salla Disease; Sialic Storage Disease; Sjögren; Sjogren-Larsson Syndrome; Van Der Knapp Disease; Vanishing White Matter Disease; Charcot-Marie-Tooth; CMT; Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency; Allan-Herndon-Dudley Syndrome; Cadasil; Cockayne Syndrome; Multiple Sulfatase Deficiency; Gangliosidoses; GM2 Gangliosidosis; BPAN; Labrune Syndrome; LCC; Mucopolysaccharidoses; TBCK-Related Intellectual Disability SyndromeLast Updated:December 18, 2023Recruiting
Condition(s):Leukodystrophy; White Matter Disease; 4H Syndrome; Adrenoleukodystrophy; AMN; ALD; ALD (Adrenoleukodystrophy); X-linked Adrenoleukodystrophy; X-ALD; Adrenomyeloneuropathy; Aicardi Goutieres Syndrome; AGS; Alexander Disease; Alexanders Leukodystrophy; AxD; ADLD; Canavan Disease; CTX; Cerebrotendinous Xanthomatoses; Krabbe Disease; GALC Deficiency; Globoid Leukodystrophy; TUBB4A-Related Leukodystrophy; H-ABC – Hypomyelination, Atrophy of Basal Ganglia and Cerebellum; HBSL; HBSL – Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity; LBSL; Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder); Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation; ALSP; CSF1R Gene Mutation; HCC – Hypomyelination and Congenital Cataract; MLC1; Megalencephalic Leukoencephalopathy With Subcortical Cysts 1; MLD; Metachromatic Leukodystrophy; PMD; Pelizaeus-Merzbacher Disease; PLP1 Null Syndrome; PLP1 Gene Duplication | Blood or Tissue | Mutations; Pelizaeus-Merzbacher-Like Disease, 1; Peroxisomal Biogenesis Disorder; Zellweger Syndrome; Refsum Disease; Salla Disease; Sialic Storage Disease; Sjögren; Sjogren-Larsson Syndrome; Van Der Knapp Disease; Vanishing White Matter Disease; Charcot-Marie-Tooth; CMT; Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency; Allan-Herndon-Dudley Syndrome; Cadasil; Cockayne Syndrome; Multiple Sulfatase Deficiency; Gangliosidoses; GM2 Gangliosidosis; BPAN; Labrune Syndrome; LCC; Mucopolysaccharidoses; TBCK-Related Intellectual Disability SyndromeLast Updated:February 2, 2024Active, not recruiting
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