Measuring Dystrophin in Dystrophinopathy Patients and Interpreting the Data
Inclement weather is expected in Washington DC and surrounding suburbs beginning early morning on March 20. If the government announces a delayed opening (2 hours for example), the meeting time will also be delayed by that amount of time. For the latest information on government closing and delays, please check the opm.gov website.
The Food and Drug Administration (FDA) is holding a public scientific workshop on March 20, 2015, to discuss dystrophin protein quantification methodologies for human tissue. This workshop is being cosponsored by the National Institutes of Health (NIH). The purpose of the workshop is to discuss currently available methodologies and to identify scientific knowledge gaps and opportunities for improving dystrophin protein detection in the context of drug development.
March 20, 2015
8:30 a.m. to 5:30 p.m.
FDA White Oak Campus
10903 New Hampshire Avenue
Bldg. 31, Room 1503A
Silver Spring, MD 20993
Registration must be received by March 17, 2015. The closing date for written and electronic comments to the division of dockets management is May 20, 2015.
For more information: please contact Mary Gross, email@example.com; or Georgiann Ienzi, Georgiann.Ienzi@fda.hhs.gov
- Glenn Morris
- Chantal Beekman
- Zarife Sahenk
- Louise Rodino-Klapac
- Jeffrey Chamberlain, PhD
- Yetrib Hathout
- Valentine Sardone
- Kathryn Wagner
- Rabi Tawil
- Detailed methodology for dystrophin quantification
- Challenges and Variability in Dystrophin Detection: Immunofluorescence, Antibody Staining and Imaging Methodologies
- Assessment of Mean Fluorescence Signal Intensity of Muscle Fibers Expressing Dystrophin
- Defining Standards for Dystrophin Quantification in DMD Muscle Biopsies
- Quantitative In Situ Measurement of Biomolecules for Companion Diagnostics
- Western blotting of dystrophin: Quantification and correlations
- Targeted Quantification of Dystrophin by Mass Spectrometry and Comparison to Antibody Based Assays
- Variable Dystrophin Content within Myofibers, between Myofibers, and between Regions of Muscles: Determining the Level of Dystrophin that makes a Clinical Impact in BMD and Manifesting Carriers of DMD
- Methods for Muscle Sampling
- MRI/MRS assessment of the status of skeletal muscle in DMD patients
- Wrap-up: Measuring Dystrophin in Patients with Dystrophinopathies and Interpreting the Data