# GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

> **NCT01784679** · — · COMPLETED · sponsor: **Ultragenyx Pharmaceutical Inc** · enrollment: 319 (actual)

## Conditions studied

- Hereditary Inclusion Body Myopathy
- GNE Myopathy
- Nonaka Disease
- Quadriceps Sparing Myopathy (QSM)
- Distal Myopathy With Rimmed Vacuoles (DMRV)

## Interventions

_None listed._

## Key facts

- **NCT ID:** NCT01784679
- **Lead sponsor:** Ultragenyx Pharmaceutical Inc
- **Sponsor class:** INDUSTRY
- **Phase:** —
- **Study type:** OBSERVATIONAL
- **Status:** COMPLETED
- **Start date:** 2013-04-05
- **Primary completion:** 2017-11-30
- **Final completion:** 2017-11-30
- **Target enrollment:** 319 (ACTUAL)
- **Last updated:** 2018-04-27

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01784679

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01784679, "GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)". Retrieved via AI Analytics 2026-06-16 from https://api.ai-analytics.org/clinical/NCT01784679. Licensed CC0.

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