Parent Project Muscular Dystrophy (PPMD) has awarded University of Minnesota researcher Dr. Rita Perlingeiro a grant for $220,000 to develop a therapeutic approach for Duchenne using a new type of "adult-derived" stem cell called "induced pluripotent stem cells" or "iPSCs." iPSCs can be created from non-controversial sources like skin, have the ability to multiply indefinitely, and can be coaxed to become many different kinds of tissue types.
Summit's Press Release:
Summit today outlines its future plans for the continued development of utrophin modulators for the treatment of DMD. Utrophin modulation is a disease modifying approach that has the potential to treat all genetic forms of DMD. Summit’s…
Added by PPMD on March 21, 2013 at 9:55am — No Comments
PTC Therapeutics, Inc. (PTC) today announced the successful completion of a $60 million financing led by Brookside Capital Partners Fund, L.P.
"This substantial financing enables PTC to continue to pursue the regulatory approval and delivery of ataluren to patients," stated Michael Schmertzler, Chairman of the Board. "PTC's Board of Directors welcomes the breadth of support and expertise of our new investors."…
Added by PPMD on March 7, 2013 at 2:00pm — No Comments
Sarepta Therapeutics has provided updates on eteplirsen and their next steps with exon skipping in their latest release: Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2012…Continue
Added by PPMD on March 7, 2013 at 1:30pm — No Comments
PPMD will provide $100,000 to Dr. Stanley Nelson of the University of California, Los Angeles, to examine the genomes of 40 boys with Duchenne who are either very mildly affected or very severely affected. The goal is to identify changes in genes other than dystrophin that may affect the course of the disease.
"We know that even boys who have exactly the same mutation in the dystrophin…
Added by PPMD on March 5, 2013 at 10:49am — No Comments
PPMD received an email from Dr. Rohit Batta, Global Medical Affairs and Patient Relations Leader in GSK's Rare Disease Unit following the recent story regarding his presentation on drisapersen at the Duchenne Parent Project Onlus meeting on February…Continue
Added by PPMD on March 4, 2013 at 11:00am — No Comments
Mary-Lou Weisman, author of Intensive Care and founder of Fund for Pete's Sake. To learn more or to read Mary-Lou's blog, visit …
Added by PPMD on March 4, 2013 at 10:30am — No Comments