Genzyme Corporation announced preliminary results from the Phase 2b clinical trial of ataluren, an investigational new drug, in patients with nonsense mutation Duchenne/Becker Muscular Dystrophy (nmDBMD). The primary endpoint of change in 6-minute walk distance did not reach statistical significance within the 48-week duration of the study. Study results showed that ataluren was well tolerated [...]
The FY2009 estimates of funding by research, condition, and disease categories were released on the NIH RePORT site yesterday. Total FY2009 funding for muscular dystrophy was $66M from the regular NIH appropriation and an additional $17M from the ARRA program. Muscular dystrophy funding level was at $56M in FY2008.
The CMD High Throughput Screen meeting at National Chemical Genomics Center (NCGC) provided the opportunity to discuss CMD targets, development and optimization of target specific assays and expanded screening to NCGC. The meeting set the goal to identify 2 CMD target assays and begin high throughput screening using NCGC resources by 2011.
The Iowa Center for Muscular Dystrophy Research seeks outstanding candidates for tenure-track positions at any rank. Successful candidates are expected to establish independent laboratories focusing on skeletal muscle biology and disease.
A postdoctoral position is available at the INSERM-UPMC-Paris VI U787-Myology Group, Institut de Myologie, Groupe Hospitalier Pitié-Salpêtrière (Paris), from March 1st 2010.
The candidates must have a Ph.D. and a strong background either in cell biology/muscle physiology or in molecular biology. Previous experience in microscopy and/or epigenetics will be an important asset.
Cure CMD announces terrific progress towards identifying treatment options in the CMDs!
Together, Cure CMD and S.A.M will fund grants to evaluate 5 promising CMD drug candidates: an NFKappaB inhibitor, laminin 111, a muscle specific IgF upregulator, N-acetyl cysteine and calcium handling compounds. A separate effort to identify disease biomarkers specific to merosin deficient CMD will also [...]
To all those that have supported Cure CMD and our efforts to find a treatment and cure Congenital Muscular Dystrophy I am very thankful. To further my involvement with finding a treatment or cure for Muscular Dystrophy, I have been offered and have accepted the position of CEO, Prothelia, Inc. I am and will continue [...]
Liestal, Switzerland, November 3, 2009 – Santhera Pharmaceuticals (SIX: SANN), a Swiss specialty pharmaceutical company focused on orphan neuromuscular diseases, announced today that the French patient advocacy organization Association Française contre les Myopathies (AFM) awarded a grant of CHF 1 million for the development of Santhera’s SNT-317 (INN: omigapil) in Congenital Muscular Dystrophy (CMD).
Ku70 which regulates Bax ( a regulator of cell apoptosis) helps improve MDC1A muscle cells and mouse
Omigapil ameliorates the pathology in MDC1A
