Search Results
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Progress Update: Optimizing the Utility of the National Registry for FSHD Research and Trial Recruitment
Jun 4, 2021, 10:58 AM
Dr. Tawil provides an update to his multi-year grant.
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Progress Update: Optimizing the Utility of the National Registry for FSHD Research and Trial Recruitment
Jun 11, 2020, 10:08 AM
Dr. Tawil provides an update on extended genetic testing for current and new patients in the National Registry.
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Progress Update: Optimizing the Utility of the National Registry for FSHD Research and Trial Recruitment
Aug 5, 2019, 10:19 AM
Dr. Tawil provides an update on year three of his project to get genetic testing performed for those in the registry who do not have it, and thus create a larger viable pool for upcoming clinical trials.
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Ask the Expert with Dr. Rabi Tawil
Aug 1, 2018, 1:00 AM
Dr. Tawil discusses his historical relationship with FSHD and the future of potential therapies.
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Progress Update: Optimizing the Utility of the National Registry for FSHD Research and Trial Recruitment
Jul 5, 2018, 11:47 AM
Dr. Tawil provides an update on year two of his project to get genetic testing performed for those in the registry who do not have it, and thus create a larger viable pool for upcoming therapeutic trials.
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Strength, Responsibility and Progress
Jun 20, 2018, 3:00 AM
Dave Lukas describes his transformation into an advocate for those with FSHD and participation in clinical trials.
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Genea Biocells Announces FDA Orphan Drug Designation for GBC0905 for the Treatment of FSHD
Jun 1, 2018, 7:48 AM
Genea Biocells announced that it has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for its therapeutic candidate, GBC0905,
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Chronic Pain in FSHD
Nov 20, 2017, 8:14 PM
Chronic Pain Has a Strong Impact on Quality of Life in FSHD.
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Progress Update: Optimizing the Utility of the National Registry for FSHD Research and Trial Recruitment
May 30, 2017, 6:07 PM
Progress update from Dr. Tawil on making the National Registry more useful for research by providing genetic testing.
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Research Opportunity
Feb 9, 2017, 2:00 PM
The University of Washington is seeking individuals to participate in a research study aimed at identifying biomarkers of FSHD.
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ENMC Workshop on FSHD Global Registries
Dec 1, 2016, 11:51 AM
Lay summary for the ENMC Workshop.
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FSHD Registry
Nov 21, 2016, 11:39 AM
Dr. Rabi Tawil discusses the National Registry based in Rochester, NY.
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aTyr Pharma ongoing Phase 1b/2 Clinical Trials
Oct 6, 2016, 9:46 AM
aTyr Pharma, a company engaged in the discovery and development of therapeutics to address severe rare diseases, is currently conducting two trials of Resolaris (ATYR1940) in patients with rare myopathies with an immune component.
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Commonality of Pain in DM & FSHD
Aug 26, 2016, 9:32 AM
258 members of the URMC Registry completed a survey last year. Results indicate that 79% of FSHD patients and 68% of DM patients experience pain.
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ENMC Travel Grant
Aug 20, 2016, 9:22 AM
Small but critical grant awarded two key stakeholders in the push for FSHD trial readiness.
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Grant Awarded to Expand FSHD Patient Registries
Apr 29, 2016, 3:56 PM
The European Neuromuscular Centre (ENMC) has awarded a grant to discuss expanding the core set of data that is collected within those registries.
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Dr. Block to Present at International FSH Research Conference
Oct 3, 2015, 1:08 PM
Dr. Block will present a short talk entitled "Global Initiatives to Accelerate FSH Research" to introduce a panel discussion with the FSHD Champions