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4 studies found for:    MYOPATHY, DISTAL, 2
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Rank Status Study
1 Completed A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy
Conditions: GNE Myopathy;   Hereditary Inclusion Body Myopathy
Interventions: Drug: Sialic Acid Extended Release (SA-ER);   Drug: Placebo
2 Recruiting A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER) Tablets in GNE Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy (HIBM)) Patients With Severe Ambulatory Impairment
Conditions: Hereditary Inclusion Body Myopathy;   Distal Myopathy With Rimmed Vacuoles;   Distal Myopathy, Nonaka Type;   GNE Myopathy;   Quadriceps Sparing Myopathy;   Inclusion Body Myopathy 2
Intervention: Drug: Aceneuramic Acid Extended-Release Tablets
3 Recruiting A Natural History Study of Patients With GNE Myopathy
Condition: Hereditary Inclusion Body Myopathy
Intervention:
4 Recruiting Survey Study and Records Review of Treatment Outcomes in Freeman-Sheldon Syndrome
Conditions: Freeman-Sheldon Syndrome;   Arthrogryposis Distal Type 2A;   Whistling Face Syndrome;   Craniocarpotarsal Dysplasia;   Craniocarpotarsal Dystrophy;   Freeman-Sheldon Syndrome Variant;   Sheldon-Hall Syndrome;   Arthrogryposis Distal Type 2B;   Gordon Syndrome;   Arthrogryposis Distal Type 3;   Arthrogryposis Distal Type 1;   Arthrogryposis, Distal, Type 1A;   Arthrogryposis Distal Type 1B;   Arthrogryposis, Distal;   Craniofacial Abnormalities;   Arthrogryposis
Interventions: Other: PTSD Checklist-Civilian (PCL-C);   Other: Modified Flanagan Quality of Life Scale;   Other: Center for Epidemiologic Studies Depression Scale (CES-D);   Other: Functional Enquiry (or Review of Systems) Form;   Other: Study of Therapeutic Outcomes and Practices in Freeman-Sheldon Syndrome (STOP-FSS) Questionnaire;   Other: FSRG Semi-Structured Quality of Life Interview (FSRG SSQLI);   Other: Medical Records Review

Study has passed its completion date and status has not been verified in more than two years.