"Have you ever wondered if anyone is examining all the data entered into DuchenneConnect? Would you like to hear the results from a study focusing on the registry data? If so, you must listen to this webinar with Dr. Stanley Nelson, Professor of Human Genetics at the David Geffen School of Medicine at the University of California, Los Angeles, as he discusses his study of the DuchenneConnect data. His research focused on analyzing the registry data from the start of the registry in 2007 through June, 2011. His research team investigated several associations, including associations between corticosteroid use, supplement use, and ambulation, and they have some very interesting results to share."

Full Credit to Parent Project Muscular Dystrophy and the Duchenne Connect Registry for bringing this information to the Duchenne Community.

BioMarin who was due to commence clinical trials for their Utrophin Upregulation drug candidate in Q1 of 2009 have announced that they are delaying the start of the trial due to "formulation and preclinical plan" related issues:

"In terms of our other preclinical programs, as announced in July of 2008, BioMarin and Summit signed a development agreement for the clinical development of SMT 1100, a small molecule inducer of utrophin, a protein that can potentially replace the defective dystrophin protein in patients with Duchenne Muscular Dystrophy.

Based on some early preclinical work, some reformulation work was needed as well as additional toxicology studies, which has pushed out the start of the clinical program. We have not yet set a time for the clinical study, but once the formulation and preclinical plan are resolved, we will be providing a more precise timeline for moving ahead to a clinical program."

Relevant Section of the Original BioMarin Q4 2008 Earnings Call Transcript