Refsum Disease: 11 Clinical Trials, Page 1 of 2

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MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Condition(s):Mucopolysaccharidosis Disorders; Hurler Syndrome; Hunter Syndrome; Maroteaux Lamy Syndrome; Sly Syndrome; Alpha-Mannosidosis; Fucosidosis; Aspartylglucosaminuria; Glycoprotein Metabolic Disorders; Sphingolipidoses; Recessive Leukodystrophies; Globoid Cell Leukodystrophy; Metachromatic Leukodystrophy; Niemann-Pick B; Niemann-Pick C Subtype 2; Sphingomyelin Deficiency; Peroxisomal Disorders; Adrenoleukodystrophy With Cerebral Involvement; Zellweger Syndrome; Neonatal Adrenoleukodystrophy; Infantile Refsum Disease; Acyl-CoA Oxidase Deficiency; D-Bifunctional Enzyme Deficiency; Multifunctional Enzyme Deficiency; Alpha-methylacyl-CoA Racmase Deficiency; Mitochondrial Neurogastrointestingal Encephalopathy; Severe Osteopetrosis; Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation); Inherited Metabolic DisordersLast Updated:November 3, 2023Recruiting

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Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD)

Condition(s):Peroxisome Biogenesis Disorder; Zellweger Spectrum Disorder; RCDP – Rhizomelic Chondrodysplasia Punctata; D-Bifunctional Protein Deficiency; Alpha-Methylacyl-CoA Racemase Deficiency; Peroxisomal Acyl-CoA Oxidase Deficiency; Peroxisomal Acyl-CoA Oxidase 2 Deficiency; ATP Binding Cassette Subfamily D Member 3 Gene Mutation; ACBD5 (AcylCoA Binding Domain 5) Deficiency; Adult Refsum Disease; Sterol Carrier Protein 2 DeficiencyLast Updated:October 31, 2023Recruiting

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The Myelin Disorders Biorepository Project

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

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Rare Disease Patient Registry & Natural History Study – Coordination of Rare Diseases at Sanford

Condition(s):Rare Disorders; Undiagnosed Disorders; Disorders of Unknown Prevalence; Cornelia De Lange Syndrome; Prenatal Benign Hypophosphatasia; Perinatal Lethal Hypophosphatasia; Odontohypophosphatasia; Adult Hypophosphatasia; Childhood-onset Hypophosphatasia; Infantile Hypophosphatasia; Hypophosphatasia; Kabuki Syndrome; Bohring-Opitz Syndrome; Narcolepsy Without Cataplexy; Narcolepsy-cataplexy; Hypersomnolence Disorder; Idiopathic Hypersomnia Without Long Sleep Time; Idiopathic Hypersomnia With Long Sleep Time; Idiopathic Hypersomnia; Kleine-Levin Syndrome; Kawasaki Disease; Leiomyosarcoma; Leiomyosarcoma of the Corpus Uteri; Leiomyosarcoma of the Cervix Uteri; Leiomyosarcoma of Small Intestine; Acquired Myasthenia Gravis; Addison Disease; Hyperacusis (Hyperacousis); Juvenile Myasthenia Gravis; Transient Neonatal Myasthenia Gravis; Williams Syndrome; Lyme Disease; Myasthenia Gravis; Marinesco Sjogren Syndrome(Marinesco-Sjogren Syndrome); Isolated Klippel-Feil Syndrome; Frasier Syndrome; Denys-Drash Syndrome; Beckwith-Wiedemann Syndrome; Emanuel Syndrome; Isolated Aniridia; Axenfeld-Rieger Syndrome; Aniridia-intellectual Disability Syndrome; Aniridia – Renal Agenesis – Psychomotor Retardation; Aniridia – Ptosis – Intellectual Disability – Familial Obesity; Aniridia – Cerebellar Ataxia – Intellectual Disability; Aniridia – Absent Patella; Aniridia; Peters Anomaly – Cataract; Peters Anomaly; Potocki-Shaffer Syndrome; Silver-Russell Syndrome Due to Maternal Uniparental Disomy of Chromosome 11; Silver-Russell Syndrome Due to Imprinting Defect of 11p15; Silver-Russell Syndrome Due to 11p15 Microduplication; Syndromic Aniridia; WAGR Syndrome; Wolf-Hirschhorn Syndrome; 4p16.3 Microduplication Syndrome; 4p Deletion Syndrome, Non-Wolf-Hirschhorn Syndrome; Autosomal Recessive Stickler Syndrome; Stickler Syndrome Type 2; Stickler Syndrome Type 1; Stickler Syndrome; Mucolipidosis Type 4; X-linked Spinocerebellar Ataxia Type 4; X-linked Spinocerebellar Ataxia Type 3; X-linked Intellectual Disability – Ataxia – Apraxia; X-linked Progressive Cerebellar Ataxia; X-linked Non Progressive Cerebellar Ataxia; X-linked Cerebellar Ataxia; Vitamin B12 Deficiency Ataxia; Toxic Exposure Ataxia; Unclassified Autosomal Dominant Spinocerebellar Ataxia; Thyroid Antibody Ataxia; Sporadic Adult-onset Ataxia of Unknown Etiology; Spinocerebellar Ataxia With Oculomotor Anomaly; Spinocerebellar Ataxia With Epilepsy; Spinocerebellar Ataxia With Axonal Neuropathy Type 2; Spinocerebellar Ataxia Type 8; Spinocerebellar Ataxia Type 7; Spinocerebellar Ataxia Type 6; Spinocerebellar Ataxia Type 5; Spinocerebellar Ataxia Type 4; Spinocerebellar Ataxia Type 37; Spinocerebellar Ataxia Type 36; Spinocerebellar Ataxia Type 35; Spinocerebellar Ataxia Type 34; Spinocerebellar Ataxia Type 32; Spinocerebellar Ataxia Type 31; Spinocerebellar Ataxia Type 30; Spinocerebellar Ataxia Type 3; Spinocerebellar Ataxia Type 29; Spinocerebellar Ataxia Type 28; Spinocerebellar Ataxia Type 27; Spinocerebellar Ataxia Type 26; Spinocerebellar Ataxia Type 25; Spinocerebellar Ataxia Type 23; Spinocerebellar Ataxia Type 22; Spinocerebellar Ataxia Type 21; Spinocerebellar Ataxia Type 20; Spinocerebellar Ataxia Type 2; Spinocerebellar Ataxia Type 19/22; Spinocerebellar Ataxia Type 18; Spinocerebellar Ataxia Type 17; Spinocerebellar Ataxia Type 16; Spinocerebellar Ataxia Type 15/16; Spinocerebellar Ataxia Type 14; Spinocerebellar Ataxia Type 13; Spinocerebellar Ataxia Type 12; Spinocerebellar Ataxia Type 11; Spinocerebellar Ataxia Type 10; Spinocerebellar Ataxia Type 1 With Axonal Neuropathy; Spinocerebellar Ataxia Type 1; Spinocerebellar Ataxia – Unknown; Spinocerebellar Ataxia – Dysmorphism; Non Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature; Spasticity-ataxia-gait Anomalies Syndrome; Spastic Ataxia With Congenital Miosis; Spastic Ataxia – Corneal Dystrophy; Spastic Ataxia; Rare Hereditary Ataxia; Rare Ataxia; Recessive Mitochondrial Ataxia Syndrome; Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature; Posterior Column Ataxia – Retinitis Pigmentosa; Post-Stroke Ataxia; Post-Head Injury Ataxia; Post Vaccination Ataxia; Polyneuropathy – Hearing Loss – Ataxia – Retinitis Pigmentosa – Cataract; Muscular Atrophy – Ataxia – Retinitis Pigmentosa – Diabetes Mellitus; Non-hereditary Degenerative Ataxia; Paroxysmal Dystonic Choreathetosis With Episodic Ataxia and Spasticity; Olivopontocerebellar Atrophy – Deafness; NARP Syndrome; Myoclonus – Cerebellar Ataxia – Deafness; Multiple System Atrophy, Parkinsonian Type; Multiple System Atrophy, Cerebellar Type; Multiple System Atrophy; Maternally-inherited Leigh Syndrome; Machado-Joseph Disease Type 3; Machado-Joseph Disease Type 2; Machado-Joseph Disease Type 1; Leigh Syndrome; Late-onset Ataxia With Dementia; Infection or Post Infection Ataxia; GAD Ataxia; Hereditary Episodic Ataxia; Gliadin/Gluten Ataxia; Friedreich Ataxia; Fragile X-associated Tremor/Ataxia Syndrome; Familial Paroxysmal Ataxia; Exposure to Medications Ataxia; Episodic Ataxia With Slurred Speech; Episodic Ataxia Unknown Type; Episodic Ataxia Type 7; Episodic Ataxia Type 6; Episodic Ataxia Type 5; Episodic Ataxia Type 4; Episodic Ataxia Type 3; Episodic Ataxia Type 1; Epilepsy and/or Ataxia With Myoclonus as Major Feature; Early-onset Spastic Ataxia-neuropathy Syndrome; Early-onset Progressive Neurodegeneration – Blindness – Ataxia – Spasticity; Early-onset Cerebellar Ataxia With Retained Tendon Reflexes; Early-onset Ataxia With Dementia; Childhood-onset Autosomal Recessive Slowly Progressive Spinocerebellar Ataxia; Dilated Cardiomyopathy With Ataxia; Cataract – Ataxia – Deafness; Cerebellar Ataxia, Cayman Type; Cerebellar Ataxia With Peripheral Neuropathy; Cerebellar Ataxia – Hypogonadism; Cerebellar Ataxia – Ectodermal Dysplasia; Cerebellar Ataxia – Areflexia – Pes Cavus – Optic Atrophy – Sensorineural Hearing Loss; Brain Tumor Ataxia; Brachydactyly – Nystagmus – Cerebellar Ataxia; Benign Paroxysmal Tonic Upgaze of Childhood With Ataxia; Autosomal Recessive Syndromic Cerebellar Ataxia; Autosomal Recessive Spastic Ataxia With Leukoencephalopathy; Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay; Autosomal Recessive Spastic Ataxia – Optic Atrophy – Dysarthria; Autosomal Recessive Spastic Ataxia; Autosomal Recessive Metabolic Cerebellar Ataxia; Autosomal Dominant Spinocerebellar Ataxia Due to Repeat Expansions That do Not Encode Polyglutamine; Autosomal Recessive Ataxia, Beauce Type; Autosomal Recessive Ataxia Due to Ubiquinone Deficiency; Autosomal Recessive Ataxia Due to PEX10 Deficiency; Autosomal Recessive Degenerative and Progressive Cerebellar Ataxia; Autosomal Recessive Congenital Cerebellar Ataxia Due to MGLUR1 Deficiency; Autosomal Recessive Congenital Cerebellar Ataxia Due to GRID2 Deficiency; Autosomal Recessive Congenital Cerebellar Ataxia; Autosomal Recessive Cerebellar Ataxia-pyramidal Signs-nystagmus-oculomotor Apraxia Syndrome; Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to WWOX Deficiency; Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to TUD Deficiency; Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to KIAA0226 Deficiency; Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome; Autosomal Recessive Cerebellar Ataxia With Late-onset Spasticity; Autosomal Recessive Cerebellar Ataxia Due to STUB1 Deficiency; Autosomal Recessive Cerebellar Ataxia Due to a DNA Repair Defect; Autosomal Recessive Cerebellar Ataxia – Saccadic Intrusion; Autosomal Recessive Cerebellar Ataxia – Psychomotor Retardation; Autosomal Recessive Cerebellar Ataxia – Blindness – Deafness; Autosomal Recessive Cerebellar Ataxia; Autosomal Dominant Spinocerebellar Ataxia Due to a Polyglutamine Anomaly; Autosomal Dominant Spinocerebellar Ataxia Due to a Point Mutation; Autosomal Dominant Spinocerebellar Ataxia Due to a Channelopathy; Autosomal Dominant Spastic Ataxia Type 1; Autosomal Dominant Spastic Ataxia; Autosomal Dominant Optic Atrophy; Ataxia-telangiectasia Variant; Ataxia-telangiectasia; Autosomal Dominant Cerebellar Ataxia, Deafness and Narcolepsy; Autosomal Dominant Cerebellar Ataxia Type 4; Autosomal Dominant Cerebellar Ataxia Type 3; Autosomal Dominant Cerebellar Ataxia Type 2; Autosomal Dominant Cerebellar Ataxia Type 1; Autosomal Dominant Cerebellar Ataxia; Ataxia-telangiectasia-like Disorder; Ataxia With Vitamin E Deficiency; Ataxia With Dementia; Ataxia – Oculomotor Apraxia Type 1; Ataxia – Other; Ataxia – Genetic Diagnosis – Unknown; Acquired Ataxia; Adult-onset Autosomal Recessive Cerebellar Ataxia; Alcohol Related Ataxia; Multiple Endocrine Neoplasia; Multiple Endocrine Neoplasia Type II; Multiple Endocrine Neoplasia Type 1; Multiple Endocrine Neoplasia Type 2; Multiple Endocrine Neoplasia, Type IV; Multiple Endocrine Neoplasia, Type 3; Multiple Endocrine Neoplasia (MEN) Syndrome; Multiple Endocrine Neoplasia Type 2B; Multiple Endocrine Neoplasia Type 2A; Atypical Hemolytic Uremic Syndrome; Atypical HUS; Wiedemann-Steiner Syndrome; Breast Implant-Associated Anaplastic Large Cell Lymphoma; Autoimmune/Inflammatory Syndrome Induced by Adjuvants (ASIA); Hemophagocytic Lymphohistiocytosis; Behcet’s Disease; Alagille Syndrome; Inclusion Body Myopathy With Early-onset Paget Disease and Frontotemporal Dementia (IBMPFD); Lowe Syndrome; Pitt Hopkins Syndrome; 1p36 Deletion Syndrome; Jansen Type Metaphyseal Chondrodysplasia; Cockayne Syndrome; Chronic Recurrent Multifocal Osteomyelitis; CRMO; Malan Syndrome; Hereditary Sensory and Autonomic Neuropathy Type Ie; VCP Disease; Hypnic Jerking; Sleep Myoclonus; Mollaret Meningitis; Recurrent Viral Meningitis; CRB1; Leber Congenital Amaurosis; Retinitis Pigmentosa; Rare Retinal Disorder; KCNMA1-Channelopathy; Primary Biliary Cirrhosis; ZMYND11; Transient Global Amnesia; Glycogen Storage Disease; Alstrom Syndrome; White Sutton Syndrome; DNM1; EIEE31; Myhre Syndrome; Recurrent Respiratory Papillomatosis; Laryngeal Papillomatosis; Tracheal Papillomatosis; Refsum Disease; Nicolaides Baraitser Syndrome; Leukodystrophy; Tango2; Cauda Equina Syndrome; Rare Gastrointestinal Disorders; Achalasia-Addisonian Syndrome; Achalasia Cardia; Achalasia Icrocephaly Syndrome; Anal Fistula; Congenital Sucrase-Isomaltase Deficiency; Eosinophilic Gastroenteritis; Idiopathic Gastroparesis; Hirschsprung Disease; Rare Inflammatory Bowel Disease; Intestinal Pseudo-Obstruction; Scleroderma; Short Bowel Syndrome; Sacral Agenesis; Sacral Agenesis Syndrome; Caudal Regression; Scheuermann Disease; SMC1A Truncated Mutations (Causing Loss of Gene Function); Cystinosis; Juvenile Nephropathic Cystinosis; Nephropathic Cystinosis; Kennedy Disease; Spinal Bulbar Muscular Atrophy; Warburg Micro Syndrome; Mucolipidoses; Mitochondrial Diseases; Mitochondrial Aminoacyl-tRNA Synthetases; Mt-aaRS Disorders; Hypertrophic Olivary Degeneration; Non-Ketotic Hyperglycinemia; Fish Odor Syndrome; Halitosis; Isolated Congenital Asplenia; Lambert Eaton (LEMS); Biliary Atresia; STAG1 Gene Mutation; Coffin Lowry Syndrome; Borjeson-Forssman-Lehman Syndrome; Blau Syndrome; Arginase 1 Deficiency; HSPB8 Myopathy; Beta-Mannosidosis; TBX4 Syndrome; DHDDS Gene Mutations; MAND-MBD5-Associated Neurodevelopmental Disorder; Constitutional Mismatch Repair Deficiency (CMMRD); SPATA5 Disorder; SPATA5L1 Related DisorderLast Updated:January 19, 2024Recruiting

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LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

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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Inherited Retinal Degenerative Disease Registry

Condition(s):Eye Diseases Hereditary; Retinal Disease; Achromatopsia; Bardet-Biedl Syndrome; Bassen-Kornzweig Syndrome; Batten Disease; Best Disease; Choroidal Dystrophy; Choroideremia; Cone Dystrophy; Cone-Rod Dystrophy; Congenital Stationary Night Blindness; Enhanced S-Cone Syndrome; Fundus Albipunctatus; Goldmann-Favre Syndrome; Gyrate Atrophy; Juvenile Macular Degeneration; Kearns-Sayre Syndrome; Leber Congenital Amaurosis; Refsum Syndrome; Retinitis Pigmentosa; Retinitis Punctata Albescens; Retinoschisis; Rod-Cone Dystrophy; Rod Dystrophy; Rod Monochromacy; Stargardt Disease; Usher SyndromeLast Updated:February 15, 2023Recruiting

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Natural History Study for Charcot Marie Tooth Disease

Condition(s):Charcot-Marie-Tooth Disease; Charcot-Marie-Tooth; Charcot-Marie-Tooth Disease, Type IA; Charcot-Marie-Tooth Disease Type 2A; Charcot-Marie-Tooth Disease Type 2; Charcot-Marie-Tooth Disease, Type 2C; Charcot-Marie-Tooth Disease Type 2A2B; Charcot-Marie-Tooth Disease Type 2B2; Charcot-Marie-Tooth Disease Type 2A1; Charcot-Marie-Tooth Disease Type 4B1; Charcot-Marie-Tooth Disease, Type IB; Charcot-Marie-Tooth Disease Type 2B1; Charcot-Marie-Tooth Disease Type 2U (Diagnosis); Charcot-Marie-Tooth Disease Type 4A; Charcot-Marie-Tooth Disease, Type 4A, Axonal Form; Charcot-Marie-Tooth Disease Type 2A2A; Charcot-Marie-Tooth Disease Type 2S (Disorder); Charcot-Marie-Tooth Disease and Deafness; Charcot-Marie-Tooth Disease Type 4B2; Charcot-Marie-Tooth Disease Type 4H; Charcot-Marie-Tooth Disease Type 1F; Charcot-Marie-Tooth Disease Type 4C; Charcot-Marie-Tooth Disease Type 4E; Charcot-Marie-Tooth Disease Type 1D; Charcot-Marie-Tooth Disease Type 2Q (Diagnosis); Charcot-Marie-Tooth Disease Type 2A2; Charcot-Marie-Tooth Disease Type 2N (Diagnosis); Charcot-Marie-Tooth Disease Type 2B5; Charcot-Marie-Tooth Disease Type 2D; Charcot-Marie-Tooth Disease Type 4D; Charcot-Marie-Tooth Disease Type 2K; Charcot-Marie-Tooth Disease Type 2L (Diagnosis); Charcot-Marie-Tooth Disease Type 2T; Charcot-Marie-Tooth Disease Type 2I; Charcot-Marie-Tooth Disease Type 2J; Charcot-Marie-Tooth Disease Type 2E; Charcot-Marie-Tooth Disease Type 2G; Charcot-Marie-Tooth Disease Type 1C; Charcot-Marie-Tooth Disease Type 2R; Charcot-Marie-Tooth Disease Type 2O (Diagnosis); Charcot-Marie-Tooth Disease Type 2M; Charcot-Marie-Tooth Disease Type 2P; Charcot-Marie-Tooth Disease Type 2Y; Charcot-Marie-Tooth Disease Type 4F (Diagnosis); Charcot-Marie-Tooth Disease Type 4B3; Charcot-Marie-Tooth Disease Type 2H; HNPP; X-Linked Charcot-Marie-Tooth DiseaseLast Updated:June 15, 2023Recruiting

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