108 studies found for:    Hereditary Neuropathies
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Rank Status Study
21 Completed RHODOS Follow-up Single-visit Study
Condition: Leber's Hereditary Optic Neuropathy
Intervention:
22 Completed Leber Hereditary Optic Neuropathy (LHON) Historical Case Record Survey
Condition: Leber Hereditary Optic Neuropathy (LHON)
Intervention:
23 Unknown  Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy
Condition: Leber Hereditary Optic Neuropathy
Intervention: Drug: cyclosporine
24 Completed Ascorbic Acid Treatment in CMT1A Trial (AATIC)
Conditions: Charcot-Marie-Tooth Disease;   Hereditary Motor and Sensory Neuropathies
Interventions: Drug: Placebo;   Drug: ascorbic acid
25 Withdrawn Study With Idebenone in Patients With Chronic Vision Loss Due to Leber's Hereditary Optic Neuropathy (LHON)
Condition: Leber's Hereditary Optic Neuropathy
Interventions: Drug: Idebenone;   Drug: Placebo
26 Not yet recruiting Post Authorisation Safety Study With Raxone in LHON Patients
Condition: Leber's Hereditary Optic Neuropathy (LHON)
Intervention: Drug: Idebenone
27 Not yet recruiting Study to Assess the Efficacy and Safety of Raxone in LHON Patients
Condition: Leber's Hereditary Optic Neuropathy (LHON)
Intervention: Drug: Idebenone
28 Completed Natural History in CCFDN and IBM Syndromes
Conditions: Inclusion Body Myositis, Sporadic;   Inclusion Body Myopathy, Autosomal-recessive;   Inclusion Body Myopathy, Autosomal-dominant;   Congenital Cataracts, Facial Dysmorphism, And Neuropathy
Intervention: Other: Natural History
29 Enrolling by invitation Open-Label, Dose-Escalating Study to Assess Safety, Tolerability, Efficacy, PK and PD of RP103 in Children With Inherited Mitochondrial Disease
Condition: Inherited Mitochondrial Disease, Including Leigh Syndrome
Intervention: Drug: Cysteamine Bitartrate Delayed-release Capsules (RP103)
30 Active, not recruiting APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis
Conditions: TTR-mediated Amyloidosis;   Amyloidosis, Hereditary;   Amyloid Neuropathies, Familial;   Familial Amyloid Polyneuropathies;   Amyloid Neuropathies;   Amyloidosis, Hereditary, Transthyretin-Related
Interventions: Drug: patisiran (ALN-TTR02);   Drug: Sterile Normal Saline (0.9% NaCl)
31 Recruiting Study of Inherited Neurological Disorders
Conditions: Ataxia;   Motor Neuron Disease;   Muscular Disease;   Muscular Dystrophy;   Peripheral Nervous System Disease
Intervention:
32 Not yet recruiting Identification of Tongue Involvement in Late-Onset Pompe Disease
Conditions: Myopathy;   Neuropathy;   Glycogen Storage Disease Type II (Late-onset Pompe Disease)
Intervention: Other: Observational study
33 Recruiting ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)
Conditions: Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC);   Amyloidosis, Hereditary;   Amyloid Neuropathies, Familial;   Amyloid Neuropathies;   Amyloidosis, Hereditary, Transthyretin-Related;   Familial Transthyretin Cardiac Amyloidosis
Interventions: Drug: Revusiran (ALN-TTRSC);   Drug: Sterile Normal Saline (0.9% NaCl)
34 Recruiting Painful Channelopathies Study
Conditions: Erythromelalgia;   Pain Insensitivity, Congenital;   Hereditary Sensory and Autonomic Neuropathies;   Chronic Pain
Intervention: Other: Observation
35 Completed Safety Study of a Single IVT Injection of QPI-1007 in Chronic Optic Nerve Atrophy and Recent Onset NAION Patients
Conditions: Optic Atrophy;   Non-arteritic Anterior Ischemic Optic Neuropathy
Intervention: Drug: QPI-1007 at various doses
36 Completed
Has Results
Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis
Condition: Familial Amyloid Polyneuropathy
Interventions: Drug: Fx-1006A;   Drug: Placebo
37 Unknown  Treadmill, Stretching and Proprioceptive Exercise (TreSPE) Rehabilitation Program for Charcot−Marie−Tooth Neuropathy Type 1A (CMT1A)
Conditions: Charcot-Marie-Tooth Disease;   Charcot-Marie-Tooth Disease Type 1A
Interventions: Other: TreSPE;   Other: SPE
38 Recruiting Stem Cell Ophthalmology Treatment Study
Conditions: Retinal Disease;   Macular Degeneration;   Hereditary Retinal Dystrophy;   Optic Nerve Disease;   Glaucoma
Interventions: Procedure: RB (Retrobulbar);   Procedure: ST (Subtenon);   Procedure: IV (Intravenous);   Procedure: IVIT (Intravitreal);   Procedure: IO (Intraocular)
39 Completed
Has Results
An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy
Conditions: Familial Amyloid Polyneuropathy;   ATTR-PN
Intervention: Drug: Fx-1006A
40 Completed Phenotype/Genotype Correlations in Neuromuscular Disorders
Condition: Neuromuscular Disease
Intervention:

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Indicates status has not been verified in more than two years