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sialic_trials [2012/05/24 20:19] – [Natural History Study] kam | sialic_trials [2012/08/27 18:41] – [Ultragenyx - HIBM Sialic Acid Phase II Trial Recruitment] daniel | ||
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Study Contact; Russ Byrns \\ | Study Contact; Russ Byrns \\ | ||
Study Coordinator | Study Coordinator | ||
+ | 310-825-3264 \\ | ||
RByrns@mednet.ucla.edu | RByrns@mednet.ucla.edu | ||
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212-263-8344 \\ | 212-263-8344 \\ | ||
Pankaj.patel@nyumc.org \\ | Pankaj.patel@nyumc.org \\ | ||
+ | |||
+ | Hadassah University Hospital \\ | ||
+ | Jerusalem, Israel, 91120 \\ | ||
+ | Principal Investigator: | ||
+ | Contact: Hannah Drory, BN, MN \\ | ||
+ | 972-2-6779398 | ||
+ | hannahd@hadassah.org.il | ||
+ | |||
To be eligible, patients must be between 18 and 65 years of age, must have a documented diagnosis of GNE myopathy (HIBM), must be able to walk 20 meters independently (may use orthotics and assistive devices), must be able to provide reproducible force in bilateral elbow flexors and knee extensors during hand-held dynamometry testing, and must be willing and able to comply with all study procedures including fine needle muscle biopsies of the upper and lower extremities at Baseline and 24 and 48 weeks. | To be eligible, patients must be between 18 and 65 years of age, must have a documented diagnosis of GNE myopathy (HIBM), must be able to walk 20 meters independently (may use orthotics and assistive devices), must be able to provide reproducible force in bilateral elbow flexors and knee extensors during hand-held dynamometry testing, and must be willing and able to comply with all study procedures including fine needle muscle biopsies of the upper and lower extremities at Baseline and 24 and 48 weeks. | ||
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* Medication history | * Medication history | ||
* Office notes from last office visit with current physician | * Office notes from last office visit with current physician | ||
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====== Natural History Study ====== | ====== Natural History Study ====== | ||
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\\ \\ | \\ \\ | ||
- | ====== HIBM Sialic Acid Trial Recruitment | + | ====== |
{{: | {{: | ||
**Ultragenyx Pharmaceutical Announces the Start of a Phase 1 Clinical Trial for Hereditary Inclusion Body Myopathy (HIBM) - also known as Nonaka Myopathy or Distal Myopathy with Rimmed Vacuoles (DMRV)** | **Ultragenyx Pharmaceutical Announces the Start of a Phase 1 Clinical Trial for Hereditary Inclusion Body Myopathy (HIBM) - also known as Nonaka Myopathy or Distal Myopathy with Rimmed Vacuoles (DMRV)** |