PTC Therapeutics ataluren webinar available online now - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

PTC Therapeutics ataluren webinar available online now - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Webinar has information on results of phase 2b study and future plans

ARTICLE POSTED: 29 JANUARY 2013 PERMANENT LINK
On January 17, 2013, Dr. Jay Barth, Vice President for Clinical Development at PTC Therapeutics hosted a webinar about the study design and timeline for ataluren development. You can watch the video recording of this on the Parent Project Muscular Dystrophy website (link below).

Research on dystrophin gene sequence could lead to treatments for Duchenne muscular dystrophy

Research on dystrophin gene sequence could lead to treatments for Duchenne muscular dystrophy: Muscular dystrophy is caused by the largest human gene, a complex chemical leviathan that has confounded scientists for decades. Research conducted at the University of Missouri and described this month in the Proceedings of the National Academy of Sciences has identified significant sections of the gene that could provide hope to young patients and families.

MU scientists Dongsheng Duan, PhD, and Yi Lai, PhD, identified a sequence in the dystrophin gene that is essential for helping muscle tissues function, a breakthrough discovery that could lead to treatments for the deadly hereditary disease. The MU researchers "found the proverbial needle in a haystack," according to Scott Harper, PhD, a muscular dystrophy expert at The Ohio State University who is not involved in the study.

Parent Project Muscular Dystrophy Grants $175,000 for Biomarkers, Non-Ambulatory Endpoints in Duchenne Muscular Dystrophy

Parent Project Muscular Dystrophy Grants $175,000 for Biomarkers, Non-Ambulatory Endpoints in Duchenne Muscular Dystrophy: HACKENSACK, N.J., Jan. 18, 2013 /PRNewswire-USNewswire/ --�Parent Project Muscular Dystrophy (PPMD) has awarded Dr. Craig McDonald of the University of California, Davis (UC Davis) $175,000 in supplemental funds to expand his ongoing study through the 20 CINRG (Cooperative International Neuromuscular Research Group) centers to better understand the progression of Duchenne muscular dystrophy (Duchenne) and determine the impact of the Duchenne standards of care established by the Centers for Disease Control (CDC).� Related studies will focus on developing new endpoints in boys who are still walking and those who can no longer walk, and identifying blood markers that track the progression of the disease.� The PPMD funding will allow Dr. McDonald to recruit 100 additional subjects into the study.

Potential stem cell therapy for Duchenne’s

Potential stem cell therapy for Duchenne’s: Researchers have found that injecting aorta-derived stem cells into the hearts of dystrophin-deficient mice prevents the onset of dilated cardiomyopathy (DCM), raising hopes of a potential new treatment approach to prevent or reverse the condition in human Duchenne muscular dystrophy.

"This is the first report of stem cell therapy yielding functional benefit in the dystrophin-deficient heart," say Suzanne Berry (University of Illinois, Urbana, USA) and team.

Tamoxifen can counteract some pathologic features in mouse model of DMD

Tamoxifen can counteract some pathologic features in mouse model of DMD: A new study has found that tamoxifen, a well-known breast cancer drug, can counteract some pathologic features in a mouse model of Duchenne muscular dystrophy (DMD). At present, no treatment is known to produce long-term improvement of the symptoms in boys with DMD, a debilitating muscular disorder that is characterized by progressive muscle wasting, respiratory and cardiac impairments, paralysis, and premature death. This study will be published in the February 2013 issue of The American Journal of Pathology.

Positive gene therapy results in large mammals of Duchenne muscular dystrophy

Positive gene therapy results in large mammals of Duchenne muscular dystrophy: Positive gene therapy results in large mammals of Duchenne muscular dystrophy