Posts by: Jose Marques Lopes, PhD

Successful results of Translarna (ataluren) and Emflaza (deflazacort) therapies for Duchenne muscular dystrophy (DMD) are highlighted in the 2017 second quarter report by PTC Therapeutics. Orally-administered Translarna is for Duchenne ... Read more

July 6, 2017July 6, 2017

Heart Failure Diagnosis and Therapy Reviewed in Duchenne Muscular Dystrophy Patients

News

End-stage heart failure is increasingly becoming the main cause of death in patients with Duchenne muscular dystrophy (DMD). Because cardiomyopathy, an abnormal heart condition, is difficult to detect, a ... Read more

May 31, 2017

Ethical Issues Raised About Proposed Database of Children with Complex Medical Problems

News

A proposed U.S. database of children with complex medical problems, including muscular dystrophy (MD), has raised concern at a respected public policy institute. Researchers at the Houston institute contend ... Read more

May 4, 2017

GTx Therapy Increases Survival, Improves Functioning, of Mice with Duchenne MD, Study Shows

News

One of a class of compounds that GTx is developing increased the survival of mice with Duchenne Muscular Dystrophy (DMD), and improved their muscle, heart and lung function, according to a pre-clinical ... Read more

April 17, 2017April 17, 2017

Bristol-Myers Squibb in Deals with Biogen, Roche to License Potential Therapies for Progressive Supranuclear Palsy, Duchenne

News

New York-based Bristol-Myers Squibb announced two separate agreements with Biogen of Cambridge, Mass., and Switzerland’s Roche to license compounds that aim to treat progressive supranuclear palsy (PSP) and Duchenne muscular dystrophy (DMD). ... Read more

April 5, 2017

Benitec’s Gene Therapy Approach Restores Muscle Strength in Mice with OPMD

News

A gene therapy approach that Benitec Biopharma is developing for oculopharyngeal muscular dystrophy (OPMD) brought impaired muscle strength in mice back to healthy levels and decreased their muscle fibrosis, ... Read more