News

The investigational treatment edasalonexent was well-tolerated, led to no safety issues, and blocked a crucial muscle disease protein in boys with Duchenne muscular dystrophy (DMD), according to part 1 results of a Phase 1/2 trial. The study, “Phase 1 Study of Edasalonexent (CAT-1004), an Oral…

A new hand-held device makes monitoring pulmonary function in adolescents with Duchenne muscular dystrophy (DMD) more convenient and may improve disease management, a study shows. The study, “Home-Based Monitoring of Pulmonary Function in Patients with Duchenne Muscular Dystrophy,” was published in the Journal of Neuromuscular…

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…

Treatment with investigational edasalonexent enabled normal growth, preserved muscle function, and slowed disease progression in boys with Duchenne muscular dystrophy (DMD), according to results of a Phase 1/2 trial and its ongoing extension study. Additionally, a Phase 3 trial called PolarisDMD (NCT03703882) also investigating edasalonexent in boys with DMD…

Citing a persistent need for new treatments and patient support, the Muscular Dystrophy Association (MDA) has updated its Duchenne muscular dystrophy (DMD) fact sheet. In related news, two top issues identified in a landmark MDA-funded survey of neuromuscular disease families were access to quality care along with a greater…

Preliminary results of a Phase 1/2 study into the gene therapy candidate SGT-001 in Duchenne muscular dystrophy (DMD) patients show low levels of microdystrophin in patients’ muscles after three months of treatment with an initial low dose and continued safety, Solid Bioscience, the therapy’s developer, reported. Continued safety was also evident,…

Sarepta Therapeutics announced that the U.S Food and Drug Administration (FDA) has accepted its application for a priority review of golodirsen (SRP-4053), a potential treatment for  Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.  A decision is expected on or around Aug. 19. Golodirsen is being tested…

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…

With the goal of connecting with every U.S. family facing a Duchenne muscular dystrophy (DMD) diagnosis, Parent Project Muscular Dystrophy (PPMD) continues its multi-year community effort called the End Duchenne Tour. At each stop, the non-profit brings updates on research, advocacy and patient care to families in…