Ironwood Pharmaceuticals, Inc., recently announced the beginning of a Phase Ib clinical trial of IW-1973 and a Phase Ia clinical trial of IW-1701, both soluble guanylate cyclase (sGC) stimulators from a wide-ranging, pharmacologically distinct portfolio of sGC composites discovered by the company. The clinical results intend to determine dose selections along with the main…
News
San Rafael, California-based biotechnology company BioMarin Pharmaceutical’s experimental drug drisapersen, which is under development as a new treatment for Duchenne muscular dystrophy (DMD), is ready to enter the market pending approval by the U.S. Food and Drug Administration (FDA). According to The Muscular Dystrophy Association (MDA) — a global…
Recent research has demonstrated that early bisphosphonate treatment of a mice model of Duchenne muscular dystrophy (DMD) had some positive effects on bone integrity and skeletal muscle strength. The research paper, entitled “Positive effects of bisphosphonates on bone and muscle in a mouse model of Duchenne muscular dystrophy,” was…
Professor Liza Pon, PhD, a leading researcher at the department of pathology and cell biology at Columbia University in New York, was recently awarded a $300,000, three-year research grant from the Muscular Dystrophy Association (MDA) to continue her work in congenital muscular dystrophy (CMD). In a recent interview conducted by…
This week, a group of interdisciplinary researchers from universities throughout Italy released study findings with potentially important clinical implications for patients with Duchenne muscular dystrophy (DMD). The study, entitled “Genetic Modifiers of Duchenne Muscular Dystrophy and Dilated Cardiomyopathy,” focused on understanding the pathophysiology of dilated cardiomyopathy (DCM), a major…
Marathon Pharmaceuticals, a company that develops new medications for rare diseases with a focus on patients who have no treatment options, recently announced that the U.S. Food and Drug Administration (FDA) has issued a rare pediatric disease designation to deflazacort to treat patients with Duchenne Muscular Dystrophy (DMD). Deflazacort is an oxazoline…
Researchers at the University of Michigan Department of Molecular & Integrative Physiology have deciphered a molecular connection leading to a potential mechanism to overcome protein dysfunction in Duchenne muscular dystrophy (DMD). The research paper, entitled “Dystrophin–glycoprotein complex regulates muscle nitric oxide production through mechanoregulation of AMPK signaling,” was…
BioBlast Pharma Ltd. has announced positive results from its Phase 2 open label clinical trial, HOPEMD, of Cabaletta (IV trehalose) in 25 patients suffering from oculopharyngeal muscular dystrophy (OPMD). OPMD is an uncommon disease in which progressive muscle-wasting occurs. People with OPMD have severe problems with swallowing (dysphagia), which can cause them…
Researchers at the University of Missouri have reported the complete and successful treatment of Duchenne muscular dystrophy (DMD) in dogs through injection of a viral vector carrying a dystrophin micro gene, leading to recovery of all muscles. Clinical trials involving humans are being planned as a next step for the team.
Parent Project Muscular Dystrophy (PPMD) announced the submission of results from its recent patient-centered benefit-risk assessment (PCBR) study to the U.S. Food & Drug Administration (FDA). PCBR studies incorporate patient assessment information and preferences in industry and regulatory decision-making. The PPMD study, developed in partnership with and sponsored by Santhera Pharmaceuticals, incorporated…
Recent Posts
- Figuring out where to buy a lift chair for my FSHD symptoms was no small task
- Sarepta plans to seek full FDA approval of 2 Duchenne treatments
- When it comes to rare disease advocacy, just keep showing up
- MDA 2026: This year’s MDA meeting provided a glimpse into the future
- I’m thankful for a big family that gives my DMD sons a full social life