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GNEM-DMP Newsletters

GNEM-DMP Newsletter - Ninth Edition
October 2017
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Previous Newsletters

GNEM-DMP Newsletter - Eighth Edition
July 2017
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GNEM-DMP Newsletter - Seventh Edition
March 2017
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GNEM-DMP Newsletter - Sixth Edition
December 2016
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GNEM-DMP Newsletter - Fifth Edition
September 2016
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GNEM-DMP Newsletter - Fourth Edition
May 2016
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The International GNE Myopathy Disease Monitoring Program (GNEM-DMP) - an ongoing longitudinal cohort study with two components, an in clinical physician reported component (sometimes referred to as Natural History Study) and an online patient reported component (also referred to as Registry).The GNE Myopathy Disease Monitoring Programme (GNEM-DMP) Registry is a global, voluntary database that will collect and report demographics, disease, and quality-of-life information directly from participants with GNE Myopathy via a secure database. The GNEM-DMP is conducted by TREAT-NMD and Ultragenyx Pharmaceutical.The GNEM-DMP Registry includes GNEM Patients from over 25 countries and is open to all GNE myopathy/Hereditary Inclusion Body Myopathy (HIBM)/Quadriceps-Sparing Myopathy (QSM)/Distal Myopathy with Rimmed Vacuoles (DMRV)/Nonaka Myopathy and IBM Type 2 patients worldwide, irrespective of treatment status. Oversight is provided to the GNEM-DMP Registry by an independent Steering Committee of international physicians, advocacy group members, TREAT-NMD and Ultragenyx representatives. The GNEM-DMP Registry will later include a portal for clinicians to access and enter data or verify data for GNE Myopathy patients under their care.The primary objectives of GNEM-DMP are to:• Identify GNE Myopathy (Hereditary Inclusion Body Myopathy (HIBM)/Quadriceps-Sparing Myopathy (QSM)/Distal Myopathy with Rimmed Vacuoles (DMRV)/Nonaka Myopathy and IBM Type 2) patients worldwide.• Promote awareness and facilitate diagnosis of GNE Myopathy, (Hereditary Inclusion Body Myopathy (HIBM)/Quadriceps-Sparing Myopathy (QSM)/Distal Myopathy with Rimmed Vacuoles (DMRV)/Nonaka Myopathy and IBM Type 2 disease) in the neuromuscular field.• Obtain an assessment of the medical history, clinical presentation and progression of disease in GNE Myopathy, (Hereditary Inclusion Body Myopathy (HIBM)/Quadriceps-Sparing Myopathy (QSM)/Distal Myopathy with Rimmed Vacuoles (DMRV)/Nonaka Myopathy and IBM Type 2 disease) patients and provide a connection for subjects to the broader GNE Myopathy community and associated programs.• Provide customized information to subjects and their physicians that desire information on their disease status and progression.You can confidentially record your GNE Myopathy, (Hereditary Inclusion Body Myopathy (HIBM)/Quadriceps-Sparing Myopathy (QSM)/Distal Myopathy with Rimmed Vacuoles (DMRV)/Nonaka Myopathy and IBM Type 2 disease) health information in the patient-reported registry of the GNEM-DMP. With global patient participation in the GNE Myopathy international patient registry, we can meet the following goals:For You: To help you track your GNE Myopathy condition, (Hereditary Inclusion Body Myopathy (HIBM)/Quadriceps-Sparing Myopathy (QSM)/Distal Myopathy with Rimmed Vacuoles (DMRV)/Nonaka Myopathy and IBM Type 2 disease) you will be able to download reports in the future to find out how you are doing and, if you choose to, compare yourself to the group of other anonymous people with GNEM in the database.For Doctors: To help doctors better manage their patients by understanding more about how GNE Myopathy, (Hereditary Inclusion Body Myopathy (HIBM)/Quadriceps-Sparing Myopathy (QSM)/Distal Myopathy with Rimmed Vacuoles (DMRV)/Nonaka Myopathy and IBM Type 2 disease) affects the body and quality of life over time.For Researchers: To help researchers design clinical trials or research studies and develop potential treatments for GNE Myopathy, (Hereditary Inclusion Body Myopathy (HIBM)/Quadriceps-Sparing Myopathy (QSM)/Distal Myopathy with Rimmed Vacuoles (DMRV)/Nonaka Myopathy and IBM Type 2 disease).For the World: To help increase awareness about this rare disease/condition and help improve the standard of care for all people with GNE Myopathy, (Hereditary Inclusion Body Myopathy (HIBM)/Quadriceps-Sparing Myopathy (QSM)/Distal Myopathy with Rimmed Vacuoles (DMRV)/Nonaka Myopathy and IBM Type 2 disease).Visit: www.gnem-dmp.com to learn more