News

Ultragenyx, a biopharma dedicated to the development of investigational products for rare diseases, will fund the efforts of a team of scientists from Saint Louis University (SLU) to advance a treatment for muscular dystrophy, inspired by lead researcher Dr. Fran Sverdrup’s daughter, who was diagnosed with the disease. SLU’s Center…

Researchers from the University of Pittsburgh recently released key findings from a study which may hold important implications for novel therapies designed to treat patients with Duchenne muscular dystrophy (DMD). The study, “Twenty-Year Follow-Up of Newborn Screening for Patients with Muscular Dystrophy,” was published in the journal…

For older boys with Duchenne muscular dystrophy (DMD), the time it takes to rise from the floor is associated with disease progression over 12 months, a new study reports. The findings, published in the journal PLOS ONE, indicate that the measure can be used as a prognostic factor…

Los Angeles-based Capricor Therapeutics has been awarded nearly $3.4 million to support its Phase 1/2 HOPE-Duchenne clinical trial. The grant is from CIRM — the California Institute for Regenerative Medicine — as part of the CIRM 2.0 program, a joint initiative meant to advance the development of treatments based on stem cells for people…

On April 9, hundreds of Texans will meet at the University of Texas Golf Club in Austin for the seventh annual Champions to CureDuchenne gala to raise both awareness for Duchenne muscular dystrophy (DMD) and funds to advance research to further the mission of CareDuchenne, which is to find a cure…

Summit Therapeutics plc announced the publication of a study into new imaging techniques that appear to reliably and reproducibly measure utrophin protein levels and muscle fiber regeneration in muscle biopsies in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) patients. The study is important to the company because one of its…

PTC Therapeutics has announced it will submit the results its Phase 3 ACT DMD clinical trial for review by Health Canada as part of the New Drug Submission (NDS) for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). DMD is a progressive disease that mostly affects male patients…

Researchers at MDI Biological Laboratory in Maine have discovered new mechanisms in heart tissue regeneration. The findings, published in the journal Development, might lead the way to drugs that help the body build new muscle cells, a crucial step toward the treatment of muscular dystrophies. Assistant Professor Voot P. Yin, who led…

Congenica, a global leader in the development of genome-based diagnostic technologies, has taken on a new project: to help a woman named Jill Viles with a serious muscle-wasting disease linked to a muscle-bound phenotype by offering to sequence her and her family’s genome. Viles and her family are affected by…

Recent advances in gene-editing technologies allow for precise manipulation of the genome to achieve therapeutic effects. While challenges remain, genome editing is likely to one day transform the treatment of muscular dystrophies, according to a review, by scientists from Editas Medicine and Duke University, that highlights current advances…