PPMD's Blog – March 2013 Archive (8)

PPMD Funds Investigator to Build New Muscle from Skin Cells to Treat Duchenne

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.

Previously, Dr.…


Added by PPMD on March 22, 2013 at 10:08am — 1 Comment

Pipeline Update: Summit outlines clinical development plans for Utrophin modulator program for Duchenne

  • First patient clinical trials of SMT C1100 expected to start H2 2013
  • Summit to showcase program at international scientific conference

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

Living Duchenne: Let's Talk About Stress, Baby

Guest post by Ivy Scherbarth. Ivy is a Colorado/Wyoming FACES Coordinator for PPMD. Follow Ivy at her blog, Living Duchenne.…


Added by PPMD on March 7, 2013 at 3:30pm — 1 Comment

Pipeline Update: Ataluren - PTC receives funds to support Phase 3 confirmatory trials

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

Pipeline Update: Eteplirsen - Sarepta's Q4 & FY12 results, latest developments, and next steps

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…


Added by PPMD on March 7, 2013 at 1:30pm — No Comments

PPMD Funds UCLA Project to Identify Genetic Changes that Affect the Severity of Duchenne

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

Pipeline Update: Drisapersen, GSK - Recent Presentation

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…


Added by PPMD on March 4, 2013 at 11:00am — No Comments

Social Isolation: A tale of two boys, and a pop quiz for you

Mary-Lou Weisman

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

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