Author Archives: Özge Özkaya, PhD

Duchenne’s Impact on Heart Reviewed, Suggesting Treatment Options

A new article reviewing the effect of Duchenne muscular dystrophy (DMD) on heart function has been published in the Journal of Neuromuscular Diseases. The article titled “Cardiac Involvement Classification and Therapeutic Management in Patients with Duchenne Muscular Dystrophy” proposes a classification for the effect of the disease on the heart and outlines the therapeutic management techniques against heart failure.

Orphan Drug Act Isn’t Healthy for Patients with Rare Diseases, Study Suggests

The current incentives offered by the U.S. Food and Drug Administration (FDA) are not efficiently stimulating orphan drug development for rare conditions like muscular dystrophy and may be benefitting pharmaceutical companies more than patients, suggest a study published in the Orphanet Journal of Rare Diseases. This is evidenced by the unmet needs of patients with such diseases and the high price of newly approved orphan drugs.

EIM Could Be Reliable Method to Assess Effects of Therapy in DMD Trials

A technique called electrical impedance myography (EIM) can help detect muscle deterioration in people with Duchenne muscular dystrophy (DMD), according to a study published in the scientific journal Annals of Neurology. The technique is also able to identify the therapeutic effects of corticosteroids and could therefore be a good biomarker in clinical trials.

Cognitive Decline Study in Late-onset DM1 Could Be Useful in Potential New Treatments

A study describing the cognitive decline over time in adults with myotonic dystrophy type 1 (DM1) was published in the scientific journal Neuromuscular Disorders. The study, titled “Cognitive decline over time in adults with myotonic dystrophy type 1: A 9-year longitudinal study” assessed the cognitive ability of the participants over a period of nine years and is the longest study to date with the largest group of participants.