Author Archives: Özge Özkaya, PhD

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.

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.

The commercialization of the recently approved drug EMFLAZA™ (deflazacort) for the treatment of Duchenne muscular dystrophy (DMD) has been paused to reconsider the pricing of the drug.

The U.S. Food and Drug Administration (FDA) yesterday announced the approval of the corticosteroid drug deflazacort (under the trade name Emflaza) for the treatment of Duchenne muscular dystrophy (DMD).

An update on the current therapeutic approaches being developed to treat Duchenne muscular dystrophy (DMD) has been published in the journal Pharmacotherapy.

Appropriate health care in Duchenne muscular dystrophy (DMD) can markedly increase quality of sleep and quality of life according to a study presenting the case of two women who are manifesting carriers of the disease.

Engagement in physical activity can increase the sense of self-worth and belonging to a group, and can help form peer relationships in children with Duchenne muscular dystrophy (MD), according to a study published in the Journal of Child Neurology.

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.

Dietary quercetin, an antioxidant belonging to group of plant pigments that gives many fruits and vegetables their color may partially protect muscles in Duchenne muscular dystrophy (MD), according to a study conducted on a mouse model of the disease and published in the scientific journal Plos One.

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.