Author Archives: Kara Elam

Monday’s announcement by the Food and Drug Administration (FDA) of accelerated approval for Exondys 51 (eteplirsen) injection, the first drug given approval for the treatment of Duchenne muscular dystrophy (DMD), was a much-celebrated likely conclusion to a debate that saw the resignation of Sarepta Therapeutics’ (makers of Exondys 51)…

A protein known as integrin may play a key role in muscle tissue growth after injury or disease, a finding that could potentially lead to therapeutic targets for muscular dystrophy (MD). The study conducted by Johns Hopkins researchers, “Targeting β1-integrin signaling enhances regeneration in aged and dystrophic muscle…

Researchers at Japan’s Kobe University Graduate School of Medicine have successfully decoded a sugar molecule, providing an important step toward understanding a mechanism linked to muscular dystrophy (MD). The study, “Identification of a Post-translational Modification with Ribitol-Phosphate and Its Defect in Muscular Dystrophy,” was published in the journal…

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…

A group of international researchers has published a review of clinical trials for a drug that shows significant potential as a therapy for people who suffer from Duchenne muscular dystrophy (DMD). The study, published in the European Neurological Review, is titled “Idebenone as a Novel Therapeutic Approach…

The Wnt signaling pathway might one day be a promising target for Duchenne muscular dystrophy (DMD) cognitive therapy, with potential clinical implications, according to a group of interdisciplinary researchers from universities throughout Chile and Australia. The team’s study, “Wnt signaling pathway improves central inhibitory synaptic transmission in a mouse model…

Researchers have treated a zebrafish animal model with a tyrosine kinase inhibitor to study its effect on fish musculature and strength in an experiment that shows promise for future therapeutic targets to treat Duchenne Muscular Dystrophy (DMD). The study, entitled “Dasatinib as a treatment for Duchenne muscular dystrophy,” was published…

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…

Massachusetts based Catabasis Pharmaceuticals,  a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics based on their proprietary pathway pharmacology technology platform, recently announced that they have begun treating patients in their MoveDMD trial, a Phase 1/2 trial of CAT- 1004 for the treatment of Duchenne…

This week researchers from the Institute for Neurodegenerative Disease, Massachusetts General Hospital (MGH), Harvard Medical School, released results from a study showing that a key protein associated with the development of Alzheimer’s disease, Amyloid precursor protein (APP), could potentially play an important role in eye and muscle health.  The study,…