News

In a recent paper published in Scientific Reports entitled “Deletion of integrin α7 subunit does not aggravate the phenotype of laminin α2 chain-deficient mice“, researchers from Sweden have revealed novel insights into the role of dystrophin related proteins towards the progression of muscular dystrophy (MD). Muscular dystrophy is…

aTyr Pharma, Inc. a company engaged in the discovery and development of Physiocrine-based therapeutics to address severe rare diseases, recently announced it began dosing the first patient in a new long term safety extension trial of Resolaris for adult patients suffering from facioscapulohumeral muscular dystrophy (FSHD), a rare and severe genetic myopathy…

Experiments that produce unexpected results are often times more informative than predictable experiments. Such is the case for a new study that found an unexpected link between the protein dystrophin and the disease atherosclerosis. A research team from the Department of Experimental Medical Science at Lund University in Sweden discovered…

Debiopharm International SA, in partnership with Solid Biosciences, recently announced the initiation of pre-clinical animal studies of Alisporivir in the treatment of Duchenne Muscular Dystrophy (DMD). DMD is a severe form of muscular dystrophy caused by a mutation in the Dystrophin gene, located in the X chromosome, which results in the absence or abnormal production of…

PTC Therapeutics recently revealed the 5 winners of its inaugural global awards program, the STRIVE Awards (Strategies to Realize Innovation, Vision and Empowerment). These awards seek to help not-for-profit organizations that cater to the Duchenne muscular dystrophy (DMD) community. PTC made the announcement during the international World Duchenne Awareness Day held on Monday,…

Researchers studying characteristics of cells isolated from muscular dystrophy patients may have found a new target to develop precision medicine. The research team found that certain molecules are increased in defective muscle fibers, and treatments that act against these molecules, such as anti-inflammatories, may be able to block their action.

The FSH Society, a nonprofit, patient-driven organization supporting research and education for facioscapulohumeral muscular dystrophy (FSHD), one of the most prevalent forms of muscular dystrophy, recently announced it will be the beneficiary of a number of fundraising events during this fall. The events which are aimed to raise awareness for the FSH…

BioMarin Pharmaceutical, Inc., a company that develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions, has officially launched kNOWyourDuchenne, a program dedicated to assisting families and clinicians gain access to genetic testing for patients with Duchenne muscular dystrophy (DMD). This first-of-its-kind program will work to pinpoint specific genetic mutations linked to…

A new study entitled “GRAF1 deficiency blunts sarcolemmal injury repair and exacerbates cardiac and skeletal muscle pathology in dystrophin-deficient mice” and published in the Skeletal Muscle Journal by Chapel Hill, North Carolina and Northwestern University in Chicago researchers, demonstrates that the GRAF1 protein aids in the repair…

A new report titled “Improving clinical trial design for Duchenne muscular dystrophy“, published in the journal BMC Neurology, suggests that some novel treatments for Duchenne muscular dystrophy (DMD) may have positive effects in those affected by the disease but these results are not showing up in clinical trials due to the way studies are being designed.