A small molecule, trichostatin A (TSA), increased the levels of a dystrophin-related protein known as utrophin, and improved the…
Articles by Patricia Inácio, PhD
GPM Investments, the sixth largest convenience store chain in the U.S., is running its “Tis the Season for Giving”…
Loss of MICU1, a key regulator of calcium balance in cells, was seen to affect muscle function and repair in…
Treatment with a cell therapy candidate called dystrophin expressing chimeras (DECs) increased dystrophin levels in heart muscle and improved cardiac…
New nine-month data from a Phase 1/2 trial show that a single administration of the gene therapy SRP-9003 (formerly, MYO-101) at low…
Due to a lack of efficacy, Acceleron Pharma is halting the clinical development of its ACE-083 candidate for the…
A review study highlights the benefits of adding an extra protein domain — called neuronal nitric oxide synthase (nNOS) —…
Twenty-five research grants totaling more than $6.6 million have been awarded by the Muscular Dystrophy Association (MDA) to support…
The U.S Food and Drug Administration (FDA) has rejected Sarepta Therapeutic’s application seeking accelerated approval of golodirsen (SRP-4053)…
Treatment with Exondys 51 (eteplirsen) significantly slows respiratory decline in boys with Duchenne muscular dystrophy (DMD), including those with more…