Thank you Jon Moulton for posting the link on Facebook, which I have incorporated here.
Orphan Drug Development in Muscular Dystrophy: Update on Two Large Clinical Trials of Dystrophin Rescue Therapies.
This paper is an insightful update on two dystrophin replacement therapies, stop codon read through and exon skipping. The article discusses the recent ataluran and drisapersen trials and outcome measures used; clinical (six minute walk test) and biochemical (dystrophin). It discusses the difficulties with the six minute walk test as an outcome measure and talks about using alternatives, such as the time to stand or the 10 meter dash. For a biochemical measure, instead of immunostaining and immunoblotting, the authors put forth a mass spec assay as a more sensitive and reliable (.99%) measure of the biomarker, dystrophin. This assay is now being independently tested.
Which brings me to the true purpose
of this post!
Yetrib Hathout, Associate
Professor, George Washington University, who developed the mass spec assay (Accurate Quantitation of Dystrophin Protein in Human Skeletal Muscle Using Mass Spectrometry) will be speaking at our Lunch and Presentation at CNMC in Washington DC along with Carsten Bonnemann,
National Institute of Neurological Disorders and Stroke, NIH, on November 19th. Time, place and directions will be reposted soon!
Message me if you want to attend (GenMedLabMom)!
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| Yetrib Hathout |
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| Carsten Bonnemann |
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